Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells.
gene delivery
gene editing
gene therapy
hematopoietic stem cells
in vivo delivery
Journal
Frontiers in genome editing
ISSN: 2673-3439
Titre abrégé: Front Genome Ed
Pays: Switzerland
ID NLM: 101775540
Informations de publication
Date de publication:
2023
2023
Historique:
received:
20
01
2023
accepted:
17
08
2023
medline:
2
10
2023
pubmed:
2
10
2023
entrez:
2
10
2023
Statut:
epublish
Résumé
Advancements in gene delivery and editing have expanded the applications of autologous hematopoietic stem and progenitor cells (HSPCs) for the treatment of monogenic and acquired diseases. The gene editing toolbox is growing, and the ability to achieve gene editing with mRNA or protein delivered intracellularly by vehicles, such as electroporation and nanoparticles, has highlighted the potential of gene editing in HSPCs. Ongoing phase I/II clinical trials with gene-edited HSPCs for β-hemoglobinopathies provide hope for treating monogenic diseases. The development of safe and efficient gene editing reagents and their delivery into hard-to-transfect HSPCs have been critical drivers in the rapid translation of HSPC gene editing into clinical studies. This review article summarizes the available payloads and delivery vehicles for gene editing HSPCs and their potential impact on therapeutic applications.
Identifiants
pubmed: 37780116
doi: 10.3389/fgeed.2023.1148693
pii: 1148693
pmc: PMC10540692
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
1148693Informations de copyright
Copyright © 2023 Murugesan, Karuppusamy, Marepally and Thangavel.
Déclaration de conflit d'intérêts
The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
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