Adenine Base Editing
Journal
GEN biotechnology
ISSN: 2768-1556
Titre abrégé: GEN Biotechnol
Pays: United States
ID NLM: 9918400388506676
Informations de publication
Date de publication:
01 Jun 2022
01 Jun 2022
Historique:
received:
29
03
2022
accepted:
11
05
2022
entrez:
11
7
2022
pubmed:
12
7
2022
medline:
12
7
2022
Statut:
ppublish
Résumé
Base editors (BEs) have opened new avenues for the treatment of genetic diseases. However, advances in delivery approaches are needed to enable disease targeting of a broad range of tissues and cell types. Adeno-associated virus (AAV) vectors remain one of the most promising delivery vehicles for gene therapies. Currently, most BE/guide combinations and their promoters exceed the packaging limit (∼5 kb) of AAVs. Dual-AAV delivery strategies often require high viral doses that impose safety concerns. In this study, we engineered an adenine base editor (ABE) using a compact Cas9 from
Identifiants
pubmed: 35811581
doi: 10.1089/genbio.2022.0015
pii: 10.1089/genbio.2022.0015
pmc: PMC9258002
doi:
Types de publication
Journal Article
Langues
eng
Pagination
285-299Subventions
Organisme : NIGMS NIH HHS
ID : R01 GM125797
Pays : United States
Informations de copyright
Copyright 2022, Mary Ann Liebert, Inc., publishers.
Déclaration de conflit d'intérêts
H.Z., N.B., P.L., X.D.G., S.A.W., W.X., and E.J.S. are coinventors on patent filings related to this study. G.G. is scientific cofounder, scientific advisor, and equity holder of Voyager Therapeutics, Adrenas Therapeutics, and Aspa Therapeutics. E.J.S. is a cofounder, scientific advisor, and equity holder of Intellia Therapeutics, and a member of the scientific advisory board of Tessera Therapeutics.
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