CRISPR medicine for blood disorders: Progress and challenges in delivery.
CRISPR/Cas
blood disorder
non-viral vectors
physical delivery
viral vectors
Journal
Frontiers in genome editing
ISSN: 2673-3439
Titre abrégé: Front Genome Ed
Pays: Switzerland
ID NLM: 101775540
Informations de publication
Date de publication:
2022
2022
Historique:
received:
05
09
2022
accepted:
22
12
2022
entrez:
23
1
2023
pubmed:
24
1
2023
medline:
24
1
2023
Statut:
epublish
Résumé
Blood disorders are a group of diseases including hematological neoplasms, clotting disorders and orphan immune deficiency diseases that affects human health. Current improvements in genome editing based therapeutics demonstrated preclinical and clinical proof to treat different blood disorders. Genome editing components such as Cas nucleases, guide RNAs and base editors are supplied in the form of either a plasmid, an mRNA, or a ribonucleoprotein complex. The most common delivery vehicles for such components include viral vectors (e.g., AAVs and RV), non-viral vectors (e.g., LNPs and polymers) and physical delivery methods (e.g., electroporation and microinjection). Each of the delivery vehicles specified above has its own advantages and disadvantages and the development of a safe transferring method for
Identifiants
pubmed: 36687779
doi: 10.3389/fgeed.2022.1037290
pii: 1037290
pmc: PMC9853164
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
1037290Informations de copyright
Copyright © 2023 Mohammadian Gol, Ureña-Bailén, Hou, Sinn, Antony, Handgretinger and Mezger.
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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