Experimental therapies for osteopetrosis.

Animals Osteopetrosis / genetics Osteoclasts / metabolism Genetic Therapy RNA Interference Therapies, Investigational
Experimental therapy Gene therapy HSCT Osteopetrosis Rare bone disease siRNA

Journal

Bone
ISSN: 1873-2763
Titre abrégé: Bone
Pays: United States
ID NLM: 8504048

Informations de publication

Date de publication:
12 2022
Historique:
received: 03 07 2022
revised: 13 09 2022
accepted: 20 09 2022
pubmed: 25 9 2022
medline: 25 10 2022
entrez: 24 9 2022
Statut: ppublish

Résumé

The medical treatment of osteopetrosis is an ongoing clinical problem. There are no effective and safer therapeutic approaches for all its forms. However, recent discoveries concerning the etiology and the pathogenesis of osteopetrosis, the development of dedicated cellular and animal models, and the advent of new technologies are paving the way for the development of targeted and safer therapies for both lethal and milder osteopetrosis. This review summarizes the huge effort and successes made by researchers to identify and develop new experimental approaches with this objective, such as the use of non-genotoxic myeloablation, gene correction of inducible Pluripotent Stem Cells (iPSCs), lentiviral-based gene therapy, protein replacement, prenatal treatment, osteoclast precursors transplantation and RNA Interference.

Identifiants

DOI: 10.1016/j.bone.2022.116567 PMID: 36152941
pubmed: 36152941
pii: S8756-3282(22)00244-7
doi: 10.1016/j.bone.2022.116567
pii:
doi:

Types de publication

Review Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

116567

Informations de copyright

Copyright © 2022 Elsevier Inc. All rights reserved.

Auteurs

Antonio Maurizi (A)

Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, 67100 L'Aquila, Italy. Electronic address: antoniomaurizi@outlook.com.

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Classifications MeSH

Recherchemedicale.com › Eucaryotes › Animaux › Experimental therapies for osteopetrosis.
Recherchemedicale.com › Maladies ostéomusculaires › Maladies osseuses › Dysplasies osseuses › Ostéosclérose › Ostéopétrose › Experimental therapies for osteopetrosis.
Recherchemedicale.com › Cellules › Cellules myéloïdes › Ostéoclastes › Experimental therapies for osteopetrosis.
Recherchemedicale.com › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Experimental therapies for osteopetrosis.
Recherchemedicale.com › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Interférence par ARN › Experimental therapies for osteopetrosis.
Recherchemedicale.com › Thérapeutique › Traitements en cours d'évaluation › Experimental therapies for osteopetrosis.