Evaluation of the Elements of Short Hairpin RNAs in Developing shRNA-Containing CAR T Cells.

C-C chemokine receptor type 5 (CCR5) Patients Living With HIV (PLWH) Rev Tat chimeric antigen receptor (CAR) hypoxanthine phosphoribosyltransferase 1 (HPRT) shRNA-containing CARs short hairpin RNA (shRNA)

Journal

Cancers
ISSN: 2072-6694
Titre abrégé: Cancers (Basel)
Pays: Switzerland
ID NLM: 101526829

Informations de publication

Date de publication:
20 May 2023
Historique:
received: 25 04 2023
revised: 16 05 2023
accepted: 18 05 2023
medline: 22 6 2023
pubmed: 22 6 2023
entrez: 22 6 2023
Statut: epublish

Résumé

Short hairpin RNAs (shRNAs) have emerged as a powerful tool for gene knockdown in various cellular systems, including chimeric antigen receptor (CAR) T cells. However, the elements of shRNAs that are crucial for their efficacy in developing shRNA-containing CAR T cells remain unclear. In this study, we evaluated the impact of different shRNA elements, including promoter strength, orientation, multiple shRNAs, self-targeting, and sense and antisense sequence composition on the knockdown efficiency of the target gene in CAR T cells. Our findings highlight the importance of considering multiple shRNAs and their orientation to achieve effective knockdown. Moreover, we demonstrate that using a strong promoter and avoiding self-targeting can enhance CAR T cell functionality. These results provide a framework for the rational design of CAR T cells with shRNA-mediated knockdown capabilities, which could improve the therapeutic efficacy of CAR T cell-based immunotherapy.

Identifiants

pubmed: 37345185
pii: cancers15102848
doi: 10.3390/cancers15102848
pmc: PMC10216594
pii:
doi:

Types de publication

Journal Article

Langues

eng

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Auteurs

Ryan Urak (R)

Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA 91010, USA.
Center for Gene Therapy, Beckman Research Institute, Duarte, CA 91010, USA.

Brenna Gittins (B)

Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA 91010, USA.

Citradewi Soemardy (C)

Center for Gene Therapy, Beckman Research Institute, Duarte, CA 91010, USA.

Nicole Grepo (N)

Center for Gene Therapy, Beckman Research Institute, Duarte, CA 91010, USA.

Lior Goldberg (L)

Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA 91010, USA.

Madeleine Maker (M)

Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA 91010, USA.

Galina Shevchenko (G)

Center for Gene Therapy, Beckman Research Institute, Duarte, CA 91010, USA.

Alicia Davis (A)

Center for Gene Therapy, Beckman Research Institute, Duarte, CA 91010, USA.

Shirley Li (S)

Center for Gene Therapy, Beckman Research Institute, Duarte, CA 91010, USA.

Tristan Scott (T)

Center for Gene Therapy, Beckman Research Institute, Duarte, CA 91010, USA.

Kevin V Morris (KV)

Menzies Health Institute Queensland, School of Pharmacy and Medical Science, Griffith University, Gold Coast Campus, Southport, QLD 4215, Australia.

Stephen J Forman (SJ)

Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA 91010, USA.

Xiuli Wang (X)

Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA 91010, USA.

Classifications MeSH