Triple vectors expand AAV transfer capacity in the retina

ABSTRACT: Maddalena et al. showed that the limited DNA transfer capacity (~4.7kb) of adeno associated viral (AAV) vectors can be expanded up to 14kb with triple AAV vectors for the efficient expression of the therapeutic CDH23 (10.1kb) and ALMS1 (12.5kb) genes.

ORGANISM(S): Homo sapiens

PROVIDER: GSE107173 | GEO | 2017/12/20

SECONDARY ACCESSION(S): PRJNA419212

REPOSITORIES: GEO