Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
AAV vector
Crigler-Najjar syndrome
UGT1A1
liver gene transfer
long-term safety
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
15 Mar 2019
15 Mar 2019
Historique:
received:
09
10
2018
accepted:
26
12
2018
entrez:
2
2
2019
pubmed:
2
2
2019
medline:
2
2
2019
Statut:
epublish
Résumé
Adeno-associated viruses (AAVs) are among the most efficient vectors for liver gene therapy. Results obtained in the first hemophilia clinical trials demonstrated the long-term efficacy of this approach in humans, showing efficient targeting of hepatocytes with both self-complementary (sc) and single-stranded (ss) AAV vectors. However, to support clinical development of AAV-based gene therapies, efficient and scalable production processes are needed. In an effort to translate to the clinic an approach of AAV-mediated liver gene transfer to treat Crigler-Najjar (CN) syndrome, we developed an (ss)AAV8 vector carrying the human UDP-glucuronosyltransferase family 1-member A1 (hUGT1A1) transgene under the control of a liver-specific promoter. We compared our construct with similar (sc)AAV8 vectors expressing hUGT1A1, showing comparable potency
Identifiants
pubmed: 30705921
doi: 10.1016/j.omtm.2018.12.011
pii: S2329-0501(18)30135-9
pmc: PMC6348934
doi:
Types de publication
Journal Article
Langues
eng
Pagination
157-174Références
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