Format

Send to

Choose Destination

See 1 citation found using an alternative search:

Sci Rep. 2013;3:1832. doi: 10.1038/srep01832.

Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy.

Author information

1
Department of Hematology, Christian Medical College, Vellore, Tamil Nadu, India.

Abstract

Despite significant advancements with recombinant AAV2 or AAV8 vectors for liver directed gene therapy in humans, it is well-recognized that host and vector-related immune challenges need to be overcome for long-term gene transfer. To overcome these limitations, alternate AAV serotypes (1-10) are being rigorously evaluated. AAV5 is the most divergent (55% similarity vs. other serotypes) and like AAV1 vector is known to transduce liver efficiently. AAV1 and AAV5 vectors are also immunologically distinct by virtue of their low seroprevalence and minimal cross reactivity against pre-existing AAV2 neutralizing antibodies. Here, we demonstrate that targeted bio-engineering of these vectors, augment their gene expression in murine hepatocytes in vivo (up to 16-fold). These studies demonstrate the feasibility of the use of these novel AAV1 and AAV5 vectors for potential gene therapy of diseases like hemophilia.

PMID:
23665951
PMCID:
PMC3652085
DOI:
10.1038/srep01832
[Indexed for MEDLINE]
Free PMC Article

Supplemental Content

Full text links

Icon for Nature Publishing Group Icon for PubMed Central
Loading ...
Support Center