Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering.

ABSTRACT: Adeno associated vectors (AAV) have shown considerable promise to treat various genetic disorders in both preclinical and clinical settings mainly because of its safety profile. However, efficient use of AAV to deliver genes in immune-competent sites like muscles and liver requires very high doses which are associated with concomitant cellular immune response against the viral capsids leading to destruction of the transduced cells. Coupled with that, there are enough evidences that at high doses, AAV particles are subjected to increased cellular phosphorylation/uniquitination leading to proteasome mediated degradation and loss of the viral particles. The presence of preexisting immunity against AAV further adds on to the problem which is acting as a major roadblock to efficiently use it as

SUBMITTER: Sen D 

PROVIDER: S-EPMC4251935 | biostudies-literature | 2014 Nov

REPOSITORIES: biostudies-literature