Adeno-associated virus vector system controlling capsid expression improves viral quantity and quality.
iScience
; 26(4): 106487, 2023 Apr 21.
Article
de En
| MEDLINE
| ID: mdl-37096037
ABSTRACT
Adeno-associated virus (AAV) vectors are promising tools for gene therapy. The current AAV vector system produces an abundance of empty capsids that are eliminated before clinical use, leading to increased costs for gene therapy. In the present study, we established an AAV production system that regulates the timing of capsid expression using a tetracycline-dependent promoter. Tetracycline-regulating capsid expression increased viral yield and reduced empty capsids in various serotypes without altering AAV vector infectivity in vitro and in vivo. The replicase expression pattern change observed in the developed AAV vector system improved viral quantity and quality, whereas timing control of capsid expression reduced empty capsids. These findings provide a new perspective on the development of AAV vector production systems in gene therapy.
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Type d'étude:
Risk_factors_studies
Langue:
En
Journal:
IScience
Année:
2023
Type de document:
Article
Pays d'affiliation:
Japon