Viral vector-based gene therapies in the clinic.
adenovirus
adeno‐associated virus
clinical translation
clinical trials
gene
gene therapy
gene transfer
herpes simplex virus
viral vector
Journal
Bioengineering & translational medicine
ISSN: 2380-6761
Titre abrégé: Bioeng Transl Med
Pays: United States
ID NLM: 101689146
Informations de publication
Date de publication:
Jan 2022
Jan 2022
Historique:
received:
16
06
2021
revised:
04
08
2021
accepted:
11
08
2021
entrez:
26
1
2022
pubmed:
27
1
2022
medline:
27
1
2022
Statut:
epublish
Résumé
Gene therapies are currently one of the most investigated therapeutic modalities in both the preclinical and clinical settings and have shown promise in treating a diverse spectrum of diseases. Gene therapies aim at introducing a gene material in target cells and represent a promising approach to cure diseases that were thought to be incurable by conventional modalities. In many cases, a gene therapy requires a vector to deliver gene therapeutics into target cells; viral vectors are among the most widely studied vectors owing to their distinguished advantages such as outstanding transduction efficiency. With decades of development, viral vector-based gene therapies have achieved promising clinical outcomes with many products approved for treating a range of diseases including cancer, infectious diseases and monogenic diseases. In addition, a number of active clinical trials are underway to further expand their therapeutic potential. In this review, we highlight the diversity of viral vectors, review approved products, and discuss the current clinical landscape of in vivo viral vector-based gene therapies. We have reviewed 13 approved products and their clinical applications. We have also analyzed more than 200 active trials based on various viral vectors and discussed their respective therapeutic applications. Moreover, we provide a critical analysis of the major translational challenges for in vivo viral vector-based gene therapies and discuss possible strategies to address the same.
Identifiants
pubmed: 35079633
doi: 10.1002/btm2.10258
pii: BTM210258
pmc: PMC8780015
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
e10258Informations de copyright
© 2021 The Authors. Bioengineering & Translational Medicine published by Wiley Periodicals LLC on behalf of American Institute of Chemical Engineers.
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