In vivo and ex vivo gene therapy for neurodegenerative diseases: a promise for disease modification.

Ababneh NA et al (2020) Correction of amyotrophic lateral sclerosis related phenotypes in induced pluripotent stem cell-derived motor neurons carrying a hexanucleotide expansion mutation in C9orf72 by CRISPR/Cas9 genome editing using homology-directed repair. Hum Mol Genet 29(13):2200–2217

pubmed: 32504093 pmcid: 7399532 doi: 10.1093/hmg/ddaa106

Abdelnour SA et al (2021) The potential of CRISPR/Cas9 gene editing as a treatment strategy for inherited diseases. Front Cell Dev Biol 9:699597

pubmed: 34977000 pmcid: 8715006 doi: 10.3389/fcell.2021.699597

Acsadi G et al (2002) Increased survival and function of SOD1 mice after glial cell-derived neurotrophic factor gene therapy. Hum Gene Ther 13(9):1047–1059

pubmed: 12067438 doi: 10.1089/104303402753812458

Alarcón-Arís D et al (2018) Selective α-synuclein knockdown in monoamine neurons by intranasal oligonucleotide delivery: potential therapy for Parkinson’s disease. Mol Ther 26(2):550–567

pubmed: 29273501 doi: 10.1016/j.ymthe.2017.11.015

Alarcón-Arís D et al (2020) Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson’s disease-like mouse model and in monkeys. EBioMedicine 59:102944

Almohaimeed HM et al (2023) Non-coding RNAs as key players in the neurodegenerative diseases: multi-platform strategies and approaches for exploring the Genome’s dark matter. J Chem Neuroanat 129:102236

Angeloni C et al (2022) Mechanisms underlying neurodegenerative disorders and potential neuroprotective activity of agrifood by-products. Antioxidants 12(1):94

pubmed: 36670956 pmcid: 9854890 doi: 10.3390/antiox12010094

Anguela XM, High KA (2019) Entering the modern era of gene therapy. Annu Rev Med 70:273–288

pubmed: 30477394 doi: 10.1146/annurev-med-012017-043332

Arias-Fuenzalida J et al (2017) FACS-assisted CRISPR-Cas9 genome editing facilitates Parkinson’s disease modeling. Stem Cell Reports 9(5):1423–1431

pubmed: 28988985 pmcid: 5830965 doi: 10.1016/j.stemcr.2017.08.026

Bäck S et al (2013) Gene therapy with AAV 2-CDNF provides functional benefits in a rat model of Parkinson’s disease. Brain and Behavior 3(2):75–88

pubmed: 23532969 pmcid: 3607149 doi: 10.1002/brb3.117

Bahmad H et al (2017) Modeling human neurological and neurodegenerative diseases: from induced pluripotent stem cells to neuronal differentiation and its applications in neurotrauma. Front Mol Neurosci 10:50

pubmed: 28293168 pmcid: 5329035 doi: 10.3389/fnmol.2017.00050

Bankiewicz KS et al (2000) Convection-enhanced delivery of AAV vector in Parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Exp Neurol 164(1):2–14

pubmed: 10877910 doi: 10.1006/exnr.2000.7408

Bankiewicz KS et al (2006) Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther 14(4):564–570

pubmed: 16829205 doi: 10.1016/j.ymthe.2006.05.005

Barker RA et al (2020) Huntingtin-lowering strategies for Huntington’s disease. Expert Opin Investig Drugs 29(10):1125–1132

pubmed: 32745442 doi: 10.1080/13543784.2020.1804552

Barros-Viegas AT et al (2020) miRNA-31 improves cognition and abolishes amyloid-β pathology by targeting APP and BACE1 in an animal model of Alzheimer’s disease. Molecular Therapy-Nucleic Acids 19:1219–1236

pubmed: 32069773 pmcid: 7031139 doi: 10.1016/j.omtn.2020.01.010

Bayarsaikhan E et al (2016) Microglial AGE-albumin is critical for neuronal death in Parkinson’s disease: a possible implication for theranostics. Int J Nanomed 10(sup1):281–292

Behrstock S et al (2006) Human neural progenitors deliver glial cell line-derived neurotrophic factor to parkinsonian rodents and aged primates. Gene Ther 13(5):379–388

pubmed: 16355116 doi: 10.1038/sj.gt.3302679

Bemelmans A-P et al (1999) Brain-derived neurotrophic factor-mediated protection of striatal neurons in an excitotoxic rat model of Huntington’s disease, as demonstrated by adenoviral gene transfer. Hum Gene Ther 10(18):2987–2997

pubmed: 10609659 doi: 10.1089/10430349950016393

Bertram L, Tanzi RE (2005) The genetic epidemiology of neurodegenerative disease. J Clin Investig 115(6):1449–1457

pubmed: 15931380 pmcid: 1137006 doi: 10.1172/JCI24761

Biju K et al (2013) Bone marrow-derived microglia-based neurturin delivery protects against dopaminergic neurodegeneration in a mouse model of Parkinson’s disease. Neurosci Lett 535:24–29

pubmed: 23295906 pmcid: 3645298 doi: 10.1016/j.neulet.2012.12.034

Blurton-Jones M et al (2014) Neural stem cells genetically-modified to express neprilysin reduce pathology in Alzheimer transgenic models. Stem Cell Res Ther 5(2):1–14

doi: 10.1186/scrt440

Borel F et al (2016) Therapeutic rAAVrh10 mediated SOD1 silencing in adult SOD1 G93A mice and nonhuman primates. Hum Gene Ther 27(1):19–31

pubmed: 26710998 doi: 10.1089/hum.2015.122

Bortolozzi A et al (2021) Oligonucleotides as therapeutic tools for brain disorders: focus on major depressive disorder and Parkinson’s disease. Pharmacol Ther 227:107873

pubmed: 33915178 doi: 10.1016/j.pharmthera.2021.107873

Bruijn LI, Miller TM, Cleveland DW (2004) Unraveling the mechanisms involved in motor neuron degeneration in ALS. Annu Rev Neurosci 27:723–749

pubmed: 15217349 doi: 10.1146/annurev.neuro.27.070203.144244

Bu L-L et al (2020) LncRNA-T199678 mitigates α-synuclein-induced dopaminergic neuron injury via miR-101-3p. Frontiers in Aging Neuroscience 12:599246

pubmed: 33328976 pmcid: 7732511 doi: 10.3389/fnagi.2020.599246

Cai L et al (2019) Downregulation of lncRNA UCA1 ameliorates the damage of dopaminergic neurons, reduces oxidative stress and inflammation in Parkinson’s disease through the inhibition of the PI3K/Akt signaling pathway. Int Immunopharmacol 75:105734

pubmed: 31301558 doi: 10.1016/j.intimp.2019.105734

Carroll JB et al (2011) Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene/allele-specific silencing of mutant huntingtin. Mol Ther 19(12):2178–2185

pubmed: 21971427 pmcid: 3242664 doi: 10.1038/mt.2011.201

Cederfjäll E et al (2012) Design of a single AAV vector for coexpression of TH and GCH1 to establish continuous DOPA synthesis in a rat model of Parkinson’s disease. Mol Ther 20(7):1315–1326

pubmed: 22294150 pmcid: 3392974 doi: 10.1038/mt.2012.1

Chen W, Hu Y, Ju D (2020) Gene therapy for neurodegenerative disorders: advances, insights and prospects. Acta Pharmaceutica Sinica B 10(8):1347–1359

pubmed: 32963936 pmcid: 7488363 doi: 10.1016/j.apsb.2020.01.015

Chen F et al (2021) CRISPR/Cas9-mediated CysLT1R deletion reverses synaptic failure, amyloidosis and cognitive impairment in APP/PS1 mice. Aging (albany NY) 13(5):6634

pubmed: 33591941 doi: 10.18632/aging.202501

Chen CX-Q et al (2022) Generation of homozygous PRKN, PINK1 and double PINK1/PRKN knockout cell lines from healthy induced pluripotent stem cells using CRISPR/Cas9 editing. Stem Cell Research 62:102806

pubmed: 35561458 doi: 10.1016/j.scr.2022.102806

Chi X, Gatti P, Papoian T (2017) Safety of antisense oligonucleotide and siRNA-based therapeutics. Drug Discovery Today 22(5):823–833

pubmed: 28159625 doi: 10.1016/j.drudis.2017.01.013

Cho IK et al (2019) Combination of stem cell and gene therapy ameliorates symptoms in Huntington’s disease mice. NPJ Regen Med 4(1):7

pubmed: 30937182 pmcid: 6435637 doi: 10.1038/s41536-019-0066-7

Chopade P et al (2023) Alzheimer’s and Parkinson’s disease therapies in the clinic. Bioeng Transl Med 8(1):e10367

pubmed: 36684083 doi: 10.1002/btm2.10367

Clark DP, Pazdernik NJ, McGehee M.R (2019) Chapter 23 - Plasmids. In Molecular Biology (3rd edn) Clark DP, Pazdernik NJ, McGehee MR, Editors. Academic Cell. pp 712–748

Cole T et al (2016) Snca targeted antisense oligonucleotides mediate progression of pathological deposition in alpha synuclein rodent transmission models of Parkinson’s disease (P6. 239). AAN Enterprises

Cole TA et al (2021) α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease. JCI insight 6(5):135633

Dabrowska M et al (2020) Generation of new isogenic models of Huntington’s disease using CRISPR-Cas9 technology. Int J Mol Sci 21(5):1854

pubmed: 32182692 pmcid: 7084361 doi: 10.3390/ijms21051854

Damien P, Allan DS (2015) Regenerative therapy and immune modulation using umbilical cord blood–derived cells. Biol Blood Marrow Transplant 21(9):1545–1554

pubmed: 26079441 doi: 10.1016/j.bbmt.2015.05.022

de Almeida LP et al (2001) Neuroprotective effect of a CNTF-expressing lentiviral vector in the quinolinic acid rat model of Huntington’s disease. Neurobiol Dis 8(3):433–446

pubmed: 11442352 doi: 10.1006/nbdi.2001.0388

de Fougerolles A et al (2007) Interfering with disease: a progress report on siRNA-based therapeutics. Nat Rev Drug Discov 6(6):443–453

pubmed: 17541417 pmcid: 7098199 doi: 10.1038/nrd2310

DeVos SL et al (2017) Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy. Science translational medicine 9(374) eaag0481

Dey ND et al (2010) Genetically engineered mesenchymal stem cells reduce behavioral deficits in the YAC 128 mouse model of Huntington’s disease. Behav Brain Res 214(2):193–200

pubmed: 20493905 doi: 10.1016/j.bbr.2010.05.023

Ding X-M et al (2019) Long non-coding RNA-p21 regulates MPP+-induced neuronal injury by targeting miR-625 and derepressing TRPM2 in SH-SY5Y cells. Chem Biol Interact 307:73–81

pubmed: 31004593 doi: 10.1016/j.cbi.2019.04.017

Dirren E et al (2015) SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice. Ann Clin Transl Neurol 2(2):167–184

pubmed: 25750921 pmcid: 4338957 doi: 10.1002/acn3.162

Dodart J-C et al (2005) Gene delivery of human apolipoprotein E alters brain Aβ burden in a mouse model of Alzheimer’s disease. Proc Natl Acad Sci 102(4):1211–1216

pubmed: 15657137 pmcid: 544620 doi: 10.1073/pnas.0409072102

Dodge JC et al (2010) AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice. Mol Ther 18(12):2075–2084

pubmed: 20859261 pmcid: 2997594 doi: 10.1038/mt.2010.206

Doxakis E (2021) Therapeutic antisense oligonucleotides for movement disorders. Med Res Rev 41(5):2656–2688

pubmed: 32656818 doi: 10.1002/med.21706

Drouet V et al (2009) Sustained effects of nonallele-specific Huntingtin silencing. Ann Neurol 65(3):276–285

pubmed: 19334076 doi: 10.1002/ana.21569

Duan W et al (2020) The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model. Gene Ther 27(3–4):157–169

pubmed: 31819203 doi: 10.1038/s41434-019-0116-1

Eberling J et al (2008) Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 70(21):1980–1983

pubmed: 18401019 doi: 10.1212/01.wnl.0000312381.29287.ff

Ebert AD et al (2008) Human neural progenitor cells over-expressing IGF-1 protect dopamine neurons and restore function in a rat model of Parkinson’s disease. Exp Neurol 209(1):213–223

pubmed: 18061591 doi: 10.1016/j.expneurol.2007.09.022

Ebert AD et al (2010) Ex vivo delivery of GDNF maintains motor function and prevents neuronal loss in a transgenic mouse model of Huntington’s disease. Exp Neurol 224(1):155–162

pubmed: 20227407 doi: 10.1016/j.expneurol.2010.03.005

Emborg ME et al (2008) GDNF-secreting human neural progenitor cells increase tyrosine hydroxylase and VMAT2 expression in MPTP-treated cynomolgus monkeys. Cell Transplant 17(4):383–395

pubmed: 18522241 doi: 10.3727/096368908784423300

Evers MM et al (2018) AAV5-miHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in a Huntington’s disease minipig model. Mol Ther 26(9):2163–2177

pubmed: 30007561 pmcid: 6127509 doi: 10.1016/j.ymthe.2018.06.021

Fouad GI (2019) Stem cells as a promising therapeutic approach for Alzheimer’s disease: a review. Bull Natl Res Cent 43(1):1–20

doi: 10.1186/s42269-019-0078-x

Friedrich M, Aigner A (2022) Therapeutic siRNA: state-of-the-art and future perspectives. BioDrugs 36(5):549–571

pubmed: 35997897 pmcid: 9396607 doi: 10.1007/s40259-022-00549-3

Fu A-L, Zhang X-M, Sun M-J (2005) Antisense inhibition of acetylcholinesterase gene expression for treating cognition deficit in Alzheimer’s disease model mice. Brain Res 1066(1–2):10–15

pubmed: 16337925 doi: 10.1016/j.brainres.2005.09.063

Fu X et al (2012) Construction of an oncolytic herpes simplex virus that precisely targets hepatocellular carcinoma cells. Mol Ther 20(2):339–346

pubmed: 22146341 doi: 10.1038/mt.2011.265

Gaj T et al (2017) In vivo genome editing improves motor function and extends survival in a mouse model of ALS. Science advances 3(12):eaar3952

pubmed: 29279867 pmcid: 5738228 doi: 10.1126/sciadv.aar3952

Garwood C et al (2017) astrocytes in Alzheimer’s disease and other age-associated dementias: a supporting player with a central role. Neuropathol Appl Neurobiol 43(4):281–298

pubmed: 27442752 doi: 10.1111/nan.12338

Gasmi M et al (2007) AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: long-term efficacy and tolerability of CERE-120 for Parkinson’s disease. Neurobiol Dis 27(1):67–76

pubmed: 17532642 doi: 10.1016/j.nbd.2007.04.003

Gentile G et al (2022) Dysregulated miRNAs as biomarkers and therapeutical targets in neurodegenerative diseases. J Pers Med 12(5):770

pubmed: 35629192 pmcid: 9143965 doi: 10.3390/jpm12050770

Gessler DJ et al (2019) Intravenous infusion of AAV for widespread gene delivery to the nervous system. Adeno-Associated Virus Vectors: Design and Delivery, pp 143–163

Glavaski-Joksimovic A et al (2010) Glial cell line-derived neurotrophic factor–secreting genetically modified human bone marrow-derived mesenchymal stem cells promote recovery in a rat model of Parkinson’s disease. J Neurosci Res 88(12):2669–2681

pubmed: 20544825 doi: 10.1002/jnr.22435

Gorbatyuk OS et al (2010) In vivo RNAi-mediated α-synuclein silencing induces nigrostriatal degeneration. Mol Ther 18(8):1450–1457

pubmed: 20551914 pmcid: 2927065 doi: 10.1038/mt.2010.115

Grondin R et al (2012) Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum. Brain 135(4):1197–1209

pubmed: 22252996 pmcid: 3326247 doi: 10.1093/brain/awr333

Gross SK et al (2020) Focal and dose-dependent neuroprotection in ALS mice following AAV2-neurturin delivery. Exp Neurol 323:113091

pubmed: 31678350 doi: 10.1016/j.expneurol.2019.113091

Guo X, Huang L (2012) Recent advances in nonviral vectors for gene delivery. Acc Chem Res 45(7):971–979

pubmed: 21870813 doi: 10.1021/ar200151m

György B et al (2018) CRISPR/Cas9 mediated disruption of the Swedish APP allele as a therapeutic approach for early-onset Alzheimer’s disease. Molecular Therapy-Nucleic Acids 11:429–440

pubmed: 29858078 pmcid: 5992788 doi: 10.1016/j.omtn.2018.03.007

Han L et al (2018) MicroRNA Let-7f-5p promotes bone marrow mesenchymal stem cells survival by targeting caspase-3 in Alzheimer disease model. Front Neurosci 12:333

pubmed: 29872375 pmcid: 5972183 doi: 10.3389/fnins.2018.00333

Harper SQ et al (2005) RNA interference improves motor and neuropathological abnormalities in a Huntington’s disease mouse model. Proc Natl Acad Sci 102(16):5820–5825

pubmed: 15811941 pmcid: 556303 doi: 10.1073/pnas.0501507102

He W et al (2020) Progress in systemic co-delivery of microRNAs and chemotherapeutics for cancer treatment by using lipid-based nanoparticles. Ther Deliv 11(9):591–603

pubmed: 32933403 doi: 10.4155/tde-2020-0052

Healy DG et al (2008) Phenotype, genotype, and worldwide genetic penetrance of LRRK2-associated Parkinson’s disease: a case-control study. Lancet Neurol 7(7):583–590

pubmed: 18539534 pmcid: 2832754 doi: 10.1016/S1474-4422(08)70117-0

Hefti F (1986) Nerve growth factor promotes survival of septal cholinergic neurons after fimbrial transections. J Neurosci 6(8):2155–2162

pubmed: 3746405 pmcid: 6568758 doi: 10.1523/JNEUROSCI.06-08-02155.1986

Helmschrodt C et al (2017) Polyethylenimine nanoparticle-mediated siRNA delivery to reduce α-Synuclein expression in a model of Parkinson’s disease. Molecular Therapy-Nucleic Acids 9:57–68

pubmed: 29246324 pmcid: 5602522 doi: 10.1016/j.omtn.2017.08.013

Heman-Ackah SM, Bassett AR, Wood MJA (2016) Precision modulation of neurodegenerative disease-related gene expression in human iPSC-derived neurons. Sci Rep 6(1):28420

pubmed: 27341390 pmcid: 4920027 doi: 10.1038/srep28420

Hemming ML et al (2007) Reducing amyloid plaque burden via ex vivo gene delivery of an Aβ-degrading protease: a novel therapeutic approach to Alzheimer disease. PLoS Med 4(8):e262

pubmed: 17760499 pmcid: 1952204 doi: 10.1371/journal.pmed.0040262

Henriques A et al (2011) CNS-targeted viral delivery of G-CSF in an animal model for ALS: improved efficacy and preservation of the neuromuscular unit. Mol Ther 19(2):284–292

pubmed: 21139572 doi: 10.1038/mt.2010.271

Hoban D, Howard L, Dowd E (2015) GDNF-secreting mesenchymal stem cells provide localized neuroprotection in an inflammation-driven rat model of Parkinson’s disease. Neuroscience 303:402–411

pubmed: 26166730 doi: 10.1016/j.neuroscience.2015.07.014

Huang EJ, Reichardt LF (2001) Neurotrophins: roles in neuronal development and function. Annu Rev Neurosci 24(1):677–736

pubmed: 11520916 pmcid: 2758233 doi: 10.1146/annurev.neuro.24.1.677

Hudry E, Vandenberghe LH (2019) Therapeutic AAV gene transfer to the nervous system: a clinical reality. Neuron 101(5):839–862

pubmed: 30844402 doi: 10.1016/j.neuron.2019.02.017

Hudry E et al (2013) Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain. Sci Transl Med 5(212):212ra161–212ra161

Hulshof S et al (2002) Cellular localization and expression patterns of interleukin-10, interleukin-4, and their receptors in multiple sclerosis lesions. Glia 38(1):24–35

pubmed: 11921201 doi: 10.1002/glia.10050

Hussain R et al (2018) Neurodegenerative diseases: regenerative mechanisms and novel therapeutic approaches. Brain Sci 8(9):177

pubmed: 30223579 pmcid: 6162719 doi: 10.3390/brainsci8090177

Huynh T-PV et al (2017) Age-dependent effects of apoE reduction using antisense oligonucleotides in a model of β-amyloidosis. Neuron 96(5):1013–1023. e4

Hwang D et al (2009) Intrathecal transplantation of human neural stem cells overexpressing VEGF provide behavioral improvement, disease onset delay and survival extension in transgenic ALS mice. Gene Ther 16(10):1234–1244

pubmed: 19626053 doi: 10.1038/gt.2009.80

Inoue N et al (2018) Knockdown of the mitochondria-localized protein p13 protects against experimental parkinsonism. EMBO Rep 19(3):e44860

pubmed: 29371327 pmcid: 5836091 doi: 10.15252/embr.201744860

Iwata N et al (2004) Presynaptic localization of neprilysin contributes to efficient clearance of amyloid-β peptide in mouse brain. J Neurosci 24(4):991–998

pubmed: 14749444 pmcid: 6729819 doi: 10.1523/JNEUROSCI.4792-03.2004

Jiang J et al (2016) Gain of toxicity from ALS/FTD-linked repeat expansions in C9ORF72 is alleviated by antisense oligonucleotides targeting GGGGCC-containing RNAs. Neuron 90(3):535–550

pubmed: 27112497 pmcid: 4860075 doi: 10.1016/j.neuron.2016.04.006

Jiang J, Wang Y, Deng M (2022) New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022. Front Pharmacol 13:1054006

pubmed: 36518658 pmcid: 9742490 doi: 10.3389/fphar.2022.1054006

Jiang H et al (2023) CRISPR/Cas9 system and its applications in nervous system diseases. Genes Dis 11(2):675–686

Kanninen K et al (2009) Intrahippocampal injection of a lentiviral vector expressing Nrf2 improves spatial learning in a mouse model of Alzheimer’s disease. Proc Natl Acad Sci 106(38):16505–16510

pubmed: 19805328 pmcid: 2752553 doi: 10.1073/pnas.0908397106

Kantor B et al (2018) Downregulation of SNCA expression by targeted editing of DNA methylation: a potential strategy for precision therapy in PD. Mol Ther 26(11):2638–2649

pubmed: 30266652 pmcid: 6224806 doi: 10.1016/j.ymthe.2018.08.019

Karimian A et al (2020) CRISPR/Cas9 novel therapeutic road for the treatment of neurodegenerative diseases. Life Sci 259:118165

pubmed: 32735884 doi: 10.1016/j.lfs.2020.118165

Kennea NL, Mehmet H (2002) Neural stem cells. J Pathol 197(4):536–550

pubmed: 12115869 doi: 10.1002/path.1189

Khalil IA, Harashima H (2018) An efficient PEGylated gene delivery system with improved targeting: synergism between octaarginine and a fusogenic peptide. Int J Pharm 538(1–2):179–187

pubmed: 29341911 doi: 10.1016/j.ijpharm.2018.01.007

Kim BW et al (2020) Human motor neurons with SOD1-G93A mutation generated from CRISPR/Cas9 gene-edited iPSCs develop pathological features of amyotrophic lateral sclerosis. Front Cell Neurosci 14:604171

pubmed: 33328898 pmcid: 7710664 doi: 10.3389/fncel.2020.604171

Kimura S, Harashima H (2020) Current status and challenges associated with CNS-targeted gene delivery across the BBB. Pharmaceutics 12(12):1216

pubmed: 33334049 pmcid: 7765480 doi: 10.3390/pharmaceutics12121216

Kiyota T et al (2010) CNS expression of anti-inflammatory cytokine interleukin-4 attenuates Alzheimer’s disease-like pathogenesis in APP+ PS1 bigenic mice. FASEB J 24(8):3093

pubmed: 20371618 pmcid: 2909296 doi: 10.1096/fj.10-155317

Klein RL et al (2000) NGF gene transfer to intrinsic basal forebrain neurons increases cholinergic cell size and protects from age-related, spatial memory deficits in middle-aged rats. Brain Res 875(1–2):144–151

pubmed: 10967308 doi: 10.1016/S0006-8993(00)02634-2

Kolli N et al (2017) CRISPR-Cas9 mediated gene-silencing of the mutant huntingtin gene in an in vitro model of Huntington’s disease. Int J Mol Sci 18(4):754

pubmed: 28368337 pmcid: 5412339 doi: 10.3390/ijms18040754

Konstantinidis E et al (2022) CRISPR-Cas9 treatment partially restores amyloid-β 42/40 in human fibroblasts with the Alzheimer’s disease PSEN1 M146L mutation. Molecular Therapy-Nucleic Acids 28:450–461

pubmed: 35505961 pmcid: 9043867 doi: 10.1016/j.omtn.2022.03.022

Kordasiewicz HB et al (2012) Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis. Neuron 74(6):1031–1044

pubmed: 22726834 pmcid: 3383626 doi: 10.1016/j.neuron.2012.05.009

Kordower JH et al (2000) Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson’s disease. Science 290(5492):767–773

pubmed: 11052933 doi: 10.1126/science.290.5492.767

Kordower JH et al (2006) Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys. Ann Neurol 60(6):706–715

pubmed: 17192932 doi: 10.1002/ana.21032

Koval ED et al (2013) Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice. Hum Mol Genet 22(20):4127–4135

pubmed: 23740943 pmcid: 3781640 doi: 10.1093/hmg/ddt261

Krakora D et al (2013) Synergistic effects of GDNF and VEGF on lifespan and disease progression in a familial ALS rat model. Mol Ther 21(8):1602–1610

pubmed: 23712039 pmcid: 3734670 doi: 10.1038/mt.2013.108

Krishnan G et al (2020) CRISPR deletion of the C9ORF72 promoter in ALS/FTD patient motor neurons abolishes production of dipeptide repeat proteins and rescues neurodegeneration. Acta Neuropathol 140:81–84

pubmed: 32266467 pmcid: 7300081 doi: 10.1007/s00401-020-02154-6

Kumar D et al (2022) Emerging therapeutic developments in neurodegenerative diseases: a clinical investigation. Drug Discovery Today 27(10):103305

pubmed: 35728774 doi: 10.1016/j.drudis.2022.06.005

Kumar S et al (2019) Novel MicroRNA-455–3p and its protective effects against abnormal APP processing and amyloid beta toxicity in Alzheimer’s disease. Biochim Biophys Acta Mol Basis Dis 1865(9):2428–2440

Lamptey RN et al (2022) A review of the common neurodegenerative disorders: current therapeutic approaches and the potential role of nanotherapeutics. Int J Mol Sci 23(3):1851

pubmed: 35163773 pmcid: 8837071 doi: 10.3390/ijms23031851

Laperle AH et al (2023) Human iPSC-derived neural progenitor cells secreting GDNF provide protection in rodent models of ALS and retinal degeneration. Stem Cell Reports 18(8):1629–1642

Lauzon M-A et al (2015) Growth factor treatment to overcome Alzheimer’s dysfunctional signaling. Cell Signal 27(6):1025–1038

pubmed: 25744541 doi: 10.1016/j.cellsig.2015.02.018

Lee HJ et al (2012) Human neural stem cells genetically modified to express human nerve growth factor (NGF) gene restore cognition in the mouse with ibotenic acid-induced cognitive dysfunction. Cell Transplant 21(11):2487–2496

pubmed: 22526467 doi: 10.3727/096368912X638964

Lee J et al (2019) CRISPR/Cas9 edited sRAGE-MSCs protect neuronal death in Parkinson’s disease model. Int J Stem Cells 12(1):114–124

pubmed: 30836725 pmcid: 6457706 doi: 10.15283/ijsc18110

Levivier M et al (1995) Intrastriatal implantation of fibroblasts genetically engineered to produce brain-derived neurotrophic factor prevents degeneration of dopaminergic neurons in a rat model of Parkinson’s disease. J Neurosci 15(12):7810–7820

pubmed: 8613721 pmcid: 6577965 doi: 10.1523/JNEUROSCI.15-12-07810.1995

LeWitt PA et al (2011) AAV2-GAD gene therapy for advanced Parkinson’s disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol 10(4):309–319

pubmed: 21419704 doi: 10.1016/S1474-4422(11)70039-4

Li X et al (2020a) Knockdown of long non-coding RNA TUG1 depresses apoptosis of hippocampal neurons in Alzheimer’s disease by elevating microRNA-15a and repressing ROCK1 expression. Inflamm Res 69:897–910

pubmed: 32577774 doi: 10.1007/s00011-020-01364-8

Li L et al (2020b) Neuro-protective roles of long non-coding RNA MALAT1 in Alzheimer’s disease with the involvement of the microRNA-30b/CNR1 network and the following PI3K/AKT activation. Exp Mol Pathol 117:104545

pubmed: 32976819 doi: 10.1016/j.yexmp.2020.104545

Li Y et al (2020c) Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. Biomaterials 234:119711

pubmed: 31945616 pmcid: 7035593 doi: 10.1016/j.biomaterials.2019.119711

Li H et al (2021) Co-editing PINK1 and DJ-1 genes via adeno-associated virus-delivered CRISPR/Cas9 system in adult monkey brain elicits classical Parkinsonian phenotype. Neurosci Bull 37(9):1271–1288

pubmed: 34165772 pmcid: 8423927 doi: 10.1007/s12264-021-00732-6

Li K, Wang Z (2023) lncRNA NEAT1: Key player in neurodegenerative diseases. Ageing Res Rev 86:101878

Lim CK et al (2020) Treatment of a mouse model of ALS by in vivo base editing. Mol Ther 28(4):1177–1189

pubmed: 31991108 pmcid: 7132599 doi: 10.1016/j.ymthe.2020.01.005

Lin Y-T et al (2018) APOE4 causes widespread molecular and cellular alterations associated with Alzheimer’s disease phenotypes in human iPSC-derived brain cell types. Neuron 98(6):1141–1154. e7

Liu Y, Wang D-A (2015) Viral vector-mediated transgenic cell therapy in regenerative medicine: safety of the process. Expert Opin Biol Ther 15(4):559–567

pubmed: 25528865 doi: 10.1517/14712598.2015.995086

Liu Y et al (2013) Investigation of the performance of PEG–PEI/ROCK-II-siRNA complexes for Alzheimer’s disease in vitro. Brain Res 1490:43–51

pubmed: 23103413 doi: 10.1016/j.brainres.2012.10.039

Liu T et al (2014) Attenuated ability of BACE1 to cleave the amyloid precursor protein via silencing long noncoding RNA BACE1-AS expression. Mol Med Rep 10(3):1275–1281

pubmed: 24970022 pmcid: 4121421 doi: 10.3892/mmr.2014.2351

Liu Z et al (2015) Therapeutic effects of transplantation of as-mir-937-expressing mesenchymal stem cells in murine model of Alzheimer’s disease. Cell Physiol Biochem 37(1):321–330

pubmed: 26316079 doi: 10.1159/000430356

Liu W et al (2021) Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics. Precis Clin Med 4(3):179–191

pubmed: 34541453 pmcid: 8444435 doi: 10.1093/pcmedi/pbab014

Lowery RL, Majewska AK (2010) Intracranial injection of adeno-associated viral vectors. J Vis Exp 45:e2140

Lu L et al (2005) Therapeutic benefit of TH-engineered mesenchymal stem cells for Parkinson’s disease. Brain Res Protoc 15(1):46–51

doi: 10.1016/j.brainresprot.2005.03.002

Manfredsson FP et al (2009) Tight long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector. Mol Ther 17(11):1857–1867

pubmed: 19707186 pmcid: 2835027 doi: 10.1038/mt.2009.196

Marks WJ et al (2008) Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson’s disease: an open-label, phase I trial. The Lancet Neurology 7(5):400–408

pubmed: 18387850 doi: 10.1016/S1474-4422(08)70065-6

Marr RA et al (2003) Neprilysin gene transfer reduces human amyloid pathology in transgenic mice. J Neurosci 23(6):1992–1996

pubmed: 12657655 pmcid: 6742010 doi: 10.1523/JNEUROSCI.23-06-01992.2003

Martier R, Konstantinova P (2020) Gene therapy for neurodegenerative diseases: slowing down the ticking clock. Front Neurosci 14:580179

pubmed: 33071748 pmcid: 7530328 doi: 10.3389/fnins.2020.580179

Martier R et al (2019) Artificial microRNAs targeting C9orf72 can reduce accumulation of intra-nuclear transcripts in ALS and FTD patients. Mol Ther Nucleic Acids 14:593–608

pubmed: 30776581 pmcid: 6378669 doi: 10.1016/j.omtn.2019.01.010

Martínez-Morales P et al (2013) Progress in stem cell therapy for major human neurological disorders. Stem Cell Rev Rep 9:685–699

pubmed: 23681704 doi: 10.1007/s12015-013-9443-6

McColgan P, Tabrizi SJ (2018) Huntington’s disease: a clinical review. Eur J Neurol 25(1):24–34

pubmed: 28817209 doi: 10.1111/ene.13413

McGinley LM et al (2016) Human cortical neural stem cells expressing insulin-like growth factor-I: a novel cellular therapy for Alzheimer’s disease. Stem Cells Transl Med 5(3):379–391

pubmed: 26744412 pmcid: 4807660 doi: 10.5966/sctm.2015-0103

Mehta M et al (2019) Oligonucleotide therapy: an emerging focus area for drug delivery in chronic inflammatory respiratory diseases. Chem Biol Interact 308:206–215

pubmed: 31136735 pmcid: 7094617 doi: 10.1016/j.cbi.2019.05.028

Migliore L, Coppedè F (2022) Gene–environment interactions in Alzheimer disease: the emerging role of epigenetics. Nat Rev Neurol 18(11):643–660

pubmed: 36180553 doi: 10.1038/s41582-022-00714-w

Miller TM et al (2005) Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis. Ann Neurol 57(5):773–776

pubmed: 15852369 pmcid: 1351126 doi: 10.1002/ana.20453

Miller TM et al (2013) An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. The Lancet Neurology 12(5):435–442

pubmed: 23541756 pmcid: 3712285 doi: 10.1016/S1474-4422(13)70061-9

Mingozzi F, High KA (2011) Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 12(5):341–355

pubmed: 21499295 doi: 10.1038/nrg2988

Mitchell MJ et al (2021) Engineering precision nanoparticles for drug delivery. Nat Rev Drug Discov 20(2):101–124

pubmed: 33277608 doi: 10.1038/s41573-020-0090-8

Nagahara AH et al (2009) Neuroprotective effects of brain-derived neurotrophic factor in rodent and primate models of Alzheimer’s disease. Nat Med 15(3):331–337

pubmed: 19198615 pmcid: 2838375 doi: 10.1038/nm.1912

Nakamori M et al (2019) Nucleic acid–based therapeutics for Parkinson’s disease. Neurotherapeutics 16:287–298

pubmed: 30756362 pmcid: 6554378 doi: 10.1007/s13311-019-00714-7

Naldini L (2011) Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet 12(5):301–315

pubmed: 21445084 doi: 10.1038/nrg2985

Nichols NL et al (2013) Intermittent hypoxia and stem cell implants preserve breathing capacity in a rodent model of amyotrophic lateral sclerosis. Am J Respir Crit Care Med 187(5):535–542

pubmed: 23220913 pmcid: 3733409 doi: 10.1164/rccm.201206-1072OC

Nilsson P et al (2010) Gene therapy in Alzheimer’s disease–potential for disease modification. J Cell Mol Med 14(4):741–757

pubmed: 20158567 pmcid: 3823109 doi: 10.1111/j.1582-4934.2010.01038.x

Nirale P, Paul A, Yadav KS (2020) Nanoemulsions for targeting the neurodegenerative diseases: Alzheimer’s, Parkinson’s and Prion’s. Life Sci 245:117394

pubmed: 32017870 doi: 10.1016/j.lfs.2020.117394

Noble W et al (2020) Considerations for future tau-targeted therapeutics: can they deliver? Expert Opin Drug Discov 15(3):265–267

pubmed: 31661994 doi: 10.1080/17460441.2020.1685977

Nuytemans K et al (2010) Genetic etiology of Parkinson disease associated with mutations in the SNCA, PARK2, PINK1, PARK7, and LRRK2 genes: a mutation update. Hum Mutat 31(7):763–780

pubmed: 20506312 pmcid: 3056147 doi: 10.1002/humu.21277

O’Keeffe FE et al (2008) Induction of A9 dopaminergic neurons from neural stem cells improves motor function in an animal model of Parkinson’s disease. Brain 131(3):630–641

pubmed: 18202103 doi: 10.1093/brain/awm340

Oksanen M et al (2017) PSEN1 mutant iPSC-derived model reveals severe astrocyte pathology in Alzheimer’s disease. Stem Cell Reports 9(6):1885–1897

pubmed: 29153989 pmcid: 5785689 doi: 10.1016/j.stemcr.2017.10.016

Olanow CW et al (2015) Trophic factors for Parkinson’s disease: to live or let die. Mov Disord 30(13):1715–1724

pubmed: 26769457 doi: 10.1002/mds.26426

Ortiz-Virumbrales M et al (2017) CRISPR/Cas9-correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer’s PSEN2 N141I neurons. Acta Neuropathol Commun 5:1–20

doi: 10.1186/s40478-017-0475-z

Ozawa K et al (2008) Cell and gene therapy using mesenchymal stem cells (MSCs). J Autoimmun 30(3):121–127

pubmed: 18249090 doi: 10.1016/j.jaut.2007.12.008

Paddison PJ et al (2002) Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells. Genes Dev 16(8):948–958

pubmed: 11959843 pmcid: 152352 doi: 10.1101/gad.981002

Palanimuthu D et al (2017) A novel class of thiosemicarbazones show multi-functional activity for the treatment of Alzheimer’s disease. Eur J Med Chem 139:612–632

pubmed: 28841514 doi: 10.1016/j.ejmech.2017.08.021

Palfi S et al (2014) Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson’s disease: a dose escalation, open-label, phase 1/2 trial. Lancet 383(9923):1138–1146

pubmed: 24412048 doi: 10.1016/S0140-6736(13)61939-X

Paolini Sguazzi G et al (2021) Induced pluripotent stem cells (iPSCs) and gene therapy: a new era for the treatment of neurological diseases. Int J Mol Sci 22(24):13674

pubmed: 34948465 pmcid: 8706293 doi: 10.3390/ijms222413674

Park H, Kim J (2022) Activation of melatonin receptor 1 by CRISPR-Cas9 activator ameliorates cognitive deficits in an Alzheimer’s disease mouse model. J Pineal Res 72(3):e12787

pubmed: 35133672 doi: 10.1111/jpi.12787

Park S et al (2009) Growth factor-expressing human neural progenitor cell grafts protect motor neurons but do not ameliorate motor performance and survival in ALS mice. Exp Mol Med 41(7):487–500

pubmed: 19322031 pmcid: 2721146 doi: 10.3858/emm.2009.41.7.054

Park D et al (2013) Improvement of cognitive function and physical activity of aging mice by human neural stem cells over-expressing choline acetyltransferase. Neurobiol Aging 34(11):2639–2646

pubmed: 23731954 doi: 10.1016/j.neurobiolaging.2013.04.026

Park H et al (2019) In vivo neuronal gene editing via CRISPR–Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer’s disease. Nat Neurosci 22(4):524–528

pubmed: 30858603 doi: 10.1038/s41593-019-0352-0

Pascual-Lucas M et al (2014) Insulin-like growth factor 2 reverses memory and synaptic deficits in APP transgenic mice. EMBO Mol Med 6(10):1246–1262

pubmed: 25100745 pmcid: 4287930 doi: 10.15252/emmm.201404228

Patil S et al (2019) The development of functional non-viral vectors for gene delivery. Int J Mol Sci 20(21):5491

pubmed: 31690044 pmcid: 6862238 doi: 10.3390/ijms20215491

Pereira PA et al (2016) Recombinant pre-miR-29b for Alzheimer’s disease therapeutics. Sci Rep 6(1):19946

pubmed: 26818210 pmcid: 4730146 doi: 10.1038/srep19946

Pertusa M et al (2008) Expression of GDNF transgene in astrocytes improves cognitive deficits in aged rats. Neurobiol Aging 29(9):1366–1379

pubmed: 17399854 doi: 10.1016/j.neurobiolaging.2007.02.026

Petrus-Reurer S et al (2021) Immunological considerations and challenges for regenerative cellular therapies. Commun Biol 4(1):798

pubmed: 34172826 pmcid: 8233383 doi: 10.1038/s42003-021-02237-4

Piao X et al (2022) Dual-gRNA approach with limited off-target effect corrects C9ORF72 repeat expansion in vivo. Sci Rep 12(1):5672

pubmed: 35383205 pmcid: 8983752 doi: 10.1038/s41598-022-07746-8

Piguet F et al (2021) The challenge of gene therapy for neurological diseases: strategies and tools to achieve efficient delivery to the central nervous system. Hum Gene Ther 32(7–8):349–374

pubmed: 33167739 doi: 10.1089/hum.2020.105

Pineda J et al (2007) Neuroprotection by GDNF-secreting stem cells in a Huntington’s disease model: optical neuroimage tracking of brain-grafted cells. Gene Ther 14(2):118–128

pubmed: 16943855 doi: 10.1038/sj.gt.3302847

Poewe W et al (2020) New hopes for disease modification in Parkinson’s disease. Neuropharmacology 171:108085

pubmed: 32298705 doi: 10.1016/j.neuropharm.2020.108085

Pollock K et al (2016) Human mesenchymal stem cells genetically engineered to overexpress brain-derived neurotrophic factor improve outcomes in Huntington’s disease mouse models. Mol Ther 24(5):965–977

pubmed: 26765769 pmcid: 4881765 doi: 10.1038/mt.2016.12

Prince M et al (2015) World Alzheimer report 2015. The global impact of dementia: an analysis of prevalence, incidence, cost and trends. Alzheimer's disease international, London

Querfurth HW, LaFerla FM (2010) Alzheimer’s disease. N Engl J Med 362(4):329–344

pubmed: 20107219 doi: 10.1056/NEJMra0909142

Rahman S et al (2019) CRISPR/Cas: An intriguing genomic editing tool with prospects in treating neurodegenerative diseases. in Seminars in cell & developmental biology. Elsevier

Rajan S, Kaas B (2022) Parkinson’s disease: risk factor modification and prevention. in Seminars in neurology. Thieme Medical Publishers, Inc. 333 Seventh Avenue, 18th Floor, New York, NY

Ralph GS et al (2005) Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med 11(4):429–433

pubmed: 15768029 doi: 10.1038/nm1205

Raoul C et al (2005) Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat Med 11(4):423–428

pubmed: 15768028 doi: 10.1038/nm1207

Raza C, Anjum R (2019) Parkinson’s disease: mechanisms, translational models and management strategies. Life Sci 226:77–90

pubmed: 30980848 doi: 10.1016/j.lfs.2019.03.057

Reeve A, Simcox E, Turnbull D (2014) Ageing and Parkinson’s disease: why is advancing age the biggest risk factor? Ageing Res Rev 14:19–30

pubmed: 24503004 pmcid: 3989046 doi: 10.1016/j.arr.2014.01.004

Ren X et al (2013) AAV2-mediated striatum delivery of human CDNF prevents the deterioration of midbrain dopamine neurons in a 6-hydroxydopamine induced parkinsonian rat model. Exp Neurol 248:148–156

pubmed: 23764500 doi: 10.1016/j.expneurol.2013.06.002

Revilla S et al (2014) Lenti-GDNF gene therapy protects against Alzheimer’s disease-like neuropathology in 3xTg-AD mice and MC65 cells. CNS Neurosci Ther 20(11):961–972

pubmed: 25119316 pmcid: 6493188 doi: 10.1111/cns.12312

Rezaei S et al (2023) Current therapies for neurological disorders and their limitations. Phytonutrients and Neurological Disorders. Elsevier, pp 107–130

doi: 10.1016/B978-0-12-824467-8.00009-7

Rinaldi C, Wood MJ (2018) Antisense oligonucleotides: the next frontier for treatment of neurological disorders. Nat Rev Neurol 14(1):9–21

pubmed: 29192260 doi: 10.1038/nrneurol.2017.148

Roberts TC, Langer R, Wood MJ (2020) Advances in oligonucleotide drug delivery. Nat Rev Drug Discov 19(10):673–694

pubmed: 32782413 pmcid: 7419031 doi: 10.1038/s41573-020-0075-7

Rook ME, Southwell AL (2022) Antisense oligonucleotide therapy: from design to the Huntington disease clinic. BioDrugs 36(2):105–119

pubmed: 35254632 pmcid: 8899000 doi: 10.1007/s40259-022-00519-9

Rosenberg MB et al (1988) Grafting genetically modified cells to the damaged brain: restorative effects of NGF expression. Science 242(4885):1575–1578

pubmed: 3201248 doi: 10.1126/science.3201248

Rosenberg JB et al (2018) AAVrh. 10-mediated APOE2 central nervous system gene therapy for APOE4-associated Alzheimer’s disease. Hum Gene Ther Clin Dev 29(1):24–47

pubmed: 29409358 pmcid: 5870071 doi: 10.1089/humc.2017.231

Ross CA, Tabrizi SJ (2011) Huntington’s disease: from molecular pathogenesis to clinical treatment. Lancet Neurol 10(1):83–98

pubmed: 21163446 doi: 10.1016/S1474-4422(10)70245-3

Sadeghian B et al (2020) Design, synthesis and biological activity evaluation of novel carbazole-benzylpiperidine hybrids as potential anti Alzheimer agents. J Mol Struct 1221:128793

doi: 10.1016/j.molstruc.2020.128793

Sadeghian I et al (2022a) Potential of cell-penetrating peptides (CPPs) in delivery of antiviral therapeutics and vaccines. Eur J Pharm Sci 169:106094

pubmed: 34896590 doi: 10.1016/j.ejps.2021.106094

Sadeghian I et al (2022b) Cell-penetrating peptide-mediated delivery of therapeutic peptides/proteins to manage the diseases involving oxidative stress, inflammatory response and apoptosis. J Pharm Pharmacol 74(8):1085–1116

pubmed: 35728949 doi: 10.1093/jpp/rgac038

Sadeghian I et al (2024) Potential of oligonucleotide-and protein/peptide-based therapeutics in the management of toxicant/stressor-induced diseases. Naunyn Schmiedebergs Arch Pharmacol 397:1275–1310

Sayed N et al (2022) Gene therapy: comprehensive overview and therapeutic applications. Life Sci 294:120375

pubmed: 35123997 doi: 10.1016/j.lfs.2022.120375

Schlich M et al (2017) Anionic liposomes for small interfering ribonucleic acid (siRNA) delivery to primary neuronal cells: evaluation of alpha-synuclein knockdown efficacy. Nano Res 10:3496–3508

doi: 10.1007/s12274-017-1561-z

Sengupta U, Kayed R (2022) Amyloid β, Tau, and α-Synuclein aggregates in the pathogenesis, prognosis, and therapeutics for neurodegenerative diseases. Prog Neurobiol 214:102270

pubmed: 35447272 doi: 10.1016/j.pneurobio.2022.102270

Shea Y-F et al (2016) A systematic review of familial Alzheimer’s disease: differences in presentation of clinical features among three mutated genes and potential ethnic differences. J Formos Med Assoc 115(2):67–75

pubmed: 26337232 doi: 10.1016/j.jfma.2015.08.004

Sherman LS et al (2019) Mesenchymal stem cell therapies in brain disease. in Seminars in cell & developmental biology. Elsevier

Shin JW et al (2016) Permanent inactivation of Huntington’s disease mutation by personalized allele-specific CRISPR/Cas9. Hum Mol Genet 25(20):4566–4576

pubmed: 28172889 pmcid: 6078600

Singer O et al (2005) Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model. Nat Neurosci 8(10):1343–1349

pubmed: 16136043 doi: 10.1038/nn1531

Skogen M et al (2006) Short G-rich oligonucleotides as a potential therapeutic for Huntington’s Disease. BMC Neurosci 7:1–16

doi: 10.1186/1471-2202-7-65

Smith D et al (1999) Age-associated neuronal atrophy occurs in the primate brain and is reversible by growth factor gene therapy. Proc Natl Acad Sci 96(19):10893–10898

pubmed: 10485922 pmcid: 17979 doi: 10.1073/pnas.96.19.10893

Smith RA et al (2006) Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Investig 116(8):2290–2296

pubmed: 16878173 pmcid: 1518790 doi: 10.1172/JCI25424

Smith PY et al (2015) miR-132/212 deficiency impairs tau metabolism and promotes pathological aggregation in vivo. Hum Mol Genet 24(23):6721–6735

pubmed: 26362250 pmcid: 4634376 doi: 10.1093/hmg/ddv377

Spencer B et al (2008) Long-term neprilysin gene transfer is associated with reduced levels of intracellular Abeta and behavioral improvement in APP transgenic mice. BMC Neurosci 9:1–12

doi: 10.1186/1471-2202-9-109

Statello L et al (2021) Gene regulation by long non-coding RNAs and its biological functions. Nat Rev Mol Cell Biol 22(2):96–118

pubmed: 33353982 doi: 10.1038/s41580-020-00315-9

Suzuki M et al (2008) Direct muscle delivery of GDNF with human mesenchymal stem cells improves motor neuron survival and function in a rat model of familial ALS. Mol Ther 16(12):2002–2010

pubmed: 18797452 doi: 10.1038/mt.2008.197

Svendsen CN et al (1998) A new method for the rapid and long term growth of human neural precursor cells. J Neurosci Methods 85(2):141–152

pubmed: 9874150 doi: 10.1016/S0165-0270(98)00126-5

Tabrizi SJ et al (2019) Targeting huntingtin expression in patients with Huntington’s disease. N Engl J Med 380(24):2307–2316

pubmed: 31059641 doi: 10.1056/NEJMoa1900907

Tan SH et al (2019) Emerging pathways to neurodegeneration: dissecting the critical molecular mechanisms in Alzheimer’s disease, Parkinson’s disease. Biomed Pharmacother 111:765–777

pubmed: 30612001 doi: 10.1016/j.biopha.2018.12.101

Taxman DJ et al (2010) Short hairpin RNA (shRNA): design, delivery, and assessment of gene knockdown. RNA Therapeutics: Function, Design, and Delivery 629:139–156

Temple S (2023) Advancing cell therapy for neurodegenerative diseases. Cell stem cell 30(5):512–529

Thomsen GM et al (2014) Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex. J Neurosci 34(47):15587–15600

pubmed: 25411487 pmcid: 4298650 doi: 10.1523/JNEUROSCI.2037-14.2014

Tu Z et al (2015) CRISPR/Cas9: a powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases. Mol Neurodegener 10:1–8

doi: 10.1186/s13024-015-0031-x

Tuszynski MH et al (1998) Targeted intraparenchymal delivery of human NGF by gene transfer to the primate basal forebrain for 3 months does not accelerate β-amyloid plaque deposition. Exp Neurol 154(2):573–582

pubmed: 9878192 doi: 10.1006/exnr.1998.6956

Tuszynski MH et al (2015) Nerve growth factor gene therapy: activation of neuronal responses in Alzheimer disease. JAMA Neurol 72(10):1139–1147

pubmed: 26302439 pmcid: 4944824 doi: 10.1001/jamaneurol.2015.1807

Uehara T et al (2019) Amido-bridged nucleic acid (AmNA)-modified antisense oligonucleotides targeting α-synuclein as a novel therapy for Parkinson’s disease. Sci Rep 9(1):7567

pubmed: 31110191 pmcid: 6527855 doi: 10.1038/s41598-019-43772-9

Ura T, Okuda K, Shimada M (2014) Developments in viral vector-based vaccines. Vaccines 2(3):624–641

pubmed: 26344749 pmcid: 4494222 doi: 10.3390/vaccines2030624

Van Bulck M et al (2019) Novel approaches for the treatment of Alzheimer’s and Parkinson’s disease. Int J Mol Sci 20(3):719

pubmed: 30743990 pmcid: 6386829 doi: 10.3390/ijms20030719

van Zundert B, Brown RH Jr (2017) Silencing strategies for therapy of SOD1-mediated ALS. Neurosci Lett 636:32–39

pubmed: 27507699 doi: 10.1016/j.neulet.2016.07.059

van Rooij E, Purcell AL, Levin AA (2012) Developing microRNA therapeutics. Circ Res 110(3):496–507

pubmed: 22302756 doi: 10.1161/CIRCRESAHA.111.247916

Vermilyea SC et al (2020) In vitro CRISPR/Cas9-directed gene editing to model LRRK2 G2019S Parkinson’s disease in common marmosets. Sci Rep 10(1):3447

pubmed: 32103062 pmcid: 7044232 doi: 10.1038/s41598-020-60273-2

Verstraeten A, Theuns J, Van Broeckhoven C (2015) Progress in unraveling the genetic etiology of Parkinson disease in a genomic era. Trends Genet 31(3):140–149

pubmed: 25703649 doi: 10.1016/j.tig.2015.01.004

Videnovic A (2013) Treatment of Huntington disease. Curr Treat Options Neurol 15:424–438

pubmed: 23417276 pmcid: 3677041 doi: 10.1007/s11940-013-0219-8

Wang H et al (2014) Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. Hum Mol Genet 23(3):668–681

pubmed: 24108104 doi: 10.1093/hmg/ddt454

Wang L et al (2017) CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs. Protein Cell 8(5):365–378

pubmed: 28401346 pmcid: 5413600 doi: 10.1007/s13238-017-0397-3

Wang P et al (2018) Systemic delivery of BACE1 siRNA through neuron-targeted nanocomplexes for treatment of Alzheimer’s disease. J Control Release 279:220–233

pubmed: 29679667 doi: 10.1016/j.jconrel.2018.04.034

Wang M et al (2019a) MicroRNA-217/138-5p downregulation inhibits inflammatory response, oxidative stress and the induction of neuronal apoptosis in MPP+-induced SH-SY5Y cells. Am J Transl Res 11(10):6619

pubmed: 31737212 pmcid: 6834497

Wang D, Tai PW, Gao G (2019b) Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov 18(5):358–378

pubmed: 30710128 pmcid: 6927556 doi: 10.1038/s41573-019-0012-9

Warren Olanow C et al (2015) Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: a double-blind, randomized, controlled trial. Ann Neurol 78(2):248–257

pubmed: 26061140 doi: 10.1002/ana.24436

Williams PD, Kingston PA (2011) Plasmid-mediated gene therapy for cardiovascular disease. Cardiovasc Res 91(4):565–576

pubmed: 21742674 doi: 10.1093/cvr/cvr197

Wilson DM et al (2023) Hallmarks of neurodegenerative diseases. Cell 186(4):693–714

pubmed: 36803602 doi: 10.1016/j.cell.2022.12.032

Winkle M et al (2021) Noncoding RNA therapeutics—challenges and potential solutions. Nat Rev Drug Discov 20(8):629–651

pubmed: 34145432 pmcid: 8212082 doi: 10.1038/s41573-021-00219-z

Wong W (2020) Economic burden of Alzheimer disease and managed care considerations. Am J Manag Care 26(8 Suppl):S177–S183

pubmed: 32840331

Wong LS et al (2004) Effects of “second-hand” smoke on structure and function of fibroblasts, cells that are critical for tissue repair and remodeling. BMC Cell Biol 5:1–14

doi: 10.1186/1471-2121-5-13

Wu S et al (2008) Neural stem cells improve learning and memory in rats with Alzheimer’s disease. Pathobiology 75(3):186–194

pubmed: 18550916 doi: 10.1159/000124979

Wu C-C et al (2016) Gain of BDNF function in engrafted neural stem cells promotes the therapeutic potential for Alzheimer’s disease. Sci Rep 6(1):27358

pubmed: 27264956 pmcid: 4893631 doi: 10.1038/srep27358

Wu X et al (2022) Development and clinical translation of ex vivo gene therapy. Comput Struct Biotechnol J 20:2986–3003

pubmed: 35782737 pmcid: 9218169 doi: 10.1016/j.csbj.2022.06.015

Xu H, Li Z, Si J (2014) Nanocarriers in gene therapy: a review. J Biomed Nanotechnol 10(12):3483–3507

pubmed: 26000367 doi: 10.1166/jbn.2014.2044

Yan R, Vassar R (2014) Targeting the β secretase BACE1 for Alzheimer’s disease therapy. Lancet Neurol 13(3):319–329

pubmed: 24556009 pmcid: 4086426 doi: 10.1016/S1474-4422(13)70276-X

Yang S et al (2017) CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease. J Clin Investig 127(7):2719–2724

pubmed: 28628038 pmcid: 5490741 doi: 10.1172/JCI92087

Yang W et al (2020) Current and projected future economic burden of Parkinson’s disease in the US. npj Parkinson’s Disease 6(1):15

pubmed: 32665974 pmcid: 7347582 doi: 10.1038/s41531-020-0117-1

Yang S et al (2021) Long non-coding RNAs in neurodegenerative diseases. Neurochem Int 148:105096

pubmed: 34118305 doi: 10.1016/j.neuint.2021.105096

Ye T et al (2021) Efficient manipulation of gene dosage in human iPSCs using CRISPR/Cas9 nickases. Commun Biol 4(1):195

pubmed: 33580208 pmcid: 7881037 doi: 10.1038/s42003-021-01722-0

Yin H et al (2017) Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing. Nat Biotechnol 35(12):1179–1187

pubmed: 29131148 pmcid: 5901668 doi: 10.1038/nbt.4005

Yoon K-J et al (2014) Modeling a genetic risk for schizophrenia in iPSCs and mice reveals neural stem cell deficits associated with adherens junctions and polarity. Cell Stem Cell 15(1):79–91

pubmed: 24996170 pmcid: 4237009 doi: 10.1016/j.stem.2014.05.003

Yoon HH et al (2022) CRISPR-Cas9 gene editing protects from the A53T-SNCA overexpression-induced pathology of Parkinson’s disease in vivo. CRISPR J 5(1):95–108

pubmed: 35191750 doi: 10.1089/crispr.2021.0025

Yoshimoto Y et al (1995) Astrocytes retrovirally transduced with BDNF elicit behavioral improvement in a rat model of Parkinson’s disease. Brain Res 691(1–2):25–36

pubmed: 8590062 doi: 10.1016/0006-8993(95)00596-I

Zahir-Jouzdani F et al (2018) siRNA delivery for treatment of degenerative diseases, new hopes and challenges. J Drug Deliv Sci Technol 45:428–441

doi: 10.1016/j.jddst.2018.04.001

Zahra W et al (2020) The global economic impact of neurodegenerative diseases: opportunities and challenges. In: Bioeconomy for Sustainable Development, Singapore: Springer, pp 333–345

Zhang P et al (2012) Effects of lateral ventricular transplantation of bone marrow-derived mesenchymal stem cells modified with brain-derived neurotrophic factor gene on cognition in a rat model of Alzheimer’s disease. Neural Regen Res 7(4):245

pubmed: 25806063 pmcid: 4353094

Zhang Q et al (2014) Caspase-3 short hairpin RNAs: a potential therapeutic agent in neurodegeneration of aluminum-exposed animal model. Curr Alzheimer Res 11(10):961–970

pubmed: 25387335 doi: 10.2174/1567205011666141107150938

Zhang W et al (2018) Knockdown of BACE1-AS by siRNA improves memory and learning behaviors in Alzheimer’s disease animal model. Exp Ther Med 16(3):2080–2086

pubmed: 30186443 pmcid: 6122303

Zhang L et al (2020) Knockdown of long non-coding RNA AL049437 mitigates MPP+-induced neuronal injury in SH-SY5Y cells via the microRNA-205-5p/MAPK1 axis. Neurotoxicology 78:29–35

pubmed: 32057949 doi: 10.1016/j.neuro.2020.02.004

Zhang Y et al (2021a) The role of non-coding RNAs in Alzheimer’s disease: from regulated mechanism to therapeutic targets and diagnostic biomarkers. Front Aging Neurosci 13:654978

pubmed: 34276336 pmcid: 8283767 doi: 10.3389/fnagi.2021.654978

Zhang L et al (2021b) Therapeutic reversal of Huntington’s disease by in vivo self-assembled siRNAs. Brain 144(11):3421–3435

pubmed: 34918046 pmcid: 8677541 doi: 10.1093/brain/awab354

Zhang Z-Y et al (2023) TRIM11 protects against tauopathies and is down-regulated in Alzheimer’s disease. Science 381(6656):eadd6696

pubmed: 37499037 doi: 10.1126/science.add6696

Zhao HT et al (2017) LRRK2 antisense oligonucleotides ameliorate α-synuclein inclusion formation in a Parkinson’s disease mouse model. Molecular Therapy-Nucleic Acids 8:508–519

pubmed: 28918051 pmcid: 5573879 doi: 10.1016/j.omtn.2017.08.002

Zhao Z et al (2020) Targeting strategies for tissue-specific drug delivery. Cell 181(1):151–167

pubmed: 32243788 doi: 10.1016/j.cell.2020.02.001

Zharikov AD et al (2015) shRNA targeting α-synuclein prevents neurodegeneration in a Parkinson’s disease model. J Clin Investig 125(7):2721–2735

pubmed: 26075822 pmcid: 4563670 doi: 10.1172/JCI64502

Zhou X et al (2015) Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer. Cell Mol Life Sci 72:1175–1184

pubmed: 25274063 doi: 10.1007/s00018-014-1744-7

Zhou S et al (2020) Long non-coding RNA NORAD functions as a microRNA-204-5p sponge to repress the progression of Parkinson’s disease in vitro by increasing the solute carrier family 5 member 3 expression. IUBMB Life 72(9):2045–2055

pubmed: 32687247 doi: 10.1002/iub.2344

In vivo and ex vivo gene therapy for neurodegenerative diseases: a promise for disease modification.

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Pouya Ebrahimi (P)

Elham Davoudi (E)

Razieh Sadeghian (R)

Amin Zaki Zadeh (AZ)

Emran Razmi (E)

Reza Heidari (R)

Mohammad Hossein Morowvat (MH)

Issa Sadeghian (I)

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