Improved gene delivery to adult mouse spinal cord through the use of engineered hybrid adeno-associated viral serotypes.

ABSTRACT: Adeno-associated viral (AAV) vectors are often used in gene therapy for neurological disorders because of its safety profile and promising results in clinical trials. One challenge to AAV gene therapy is effective transduction of large numbers of the appropriate cell type, which can be overcome by modulating the viral capsid through DNA shuffling. Our previous study demonstrates that Rec2, among a family of novel engineered hybrid capsid serotypes (Rec1~4) transduces adipose tissue with far superior efficiency than naturally occurring AAV serotypes. Here we assessed the transduction of adult spinal cord at two different doses of AAV vectors expressing green fluorescent protein (2 × 10⁹ or 4 × 10⁸ viral particles) via intraparenchymal injection at the thoracic vertebra

SUBMITTER: Siu JJ 

PROVIDER: S-EPMC5472488 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature