scAAV1-Syn-GFP

Recombinant AAV is an efficient vector for central nervous system (CNS) gene delivery: the virus can infect postmitotic neurons and result in sustained transgene expression with little immunogenicity or toxicity. The utility of AAV has been demonstrated in numerous preclinical studies and is currently used in clinical trials for Alzheimer's disease, Parkinson's disease, Canavan disease, and Batten disease.

Self-complementary vectors have also been used in biodistribution studies aimed at delivery to CNS tissues. The ability to detect transduction even at low levels of delivery has allowed optimization of vector serotypes and injection regimens, leading to significant delivery. Studies have demonstrated significant transduction rates using scAAV vectors following intramuscular injection or direct injection into the sciatic nerve. Among serotypes 1–6, scAAV-1 pseudotyped vectors produced the highest number of transduced cells by either injection route (4.1% and 7.5% of targeted motor neurons for intramuscular and intraschiatic injections, respectively). Following intraschial injection, direct comparison of this scAAV-1 vector and the related ssAAV-1, scAAV transduced eight times as many neurons at a tenfold lower dose.