Ontology highlight
ABSTRACT:
SUBMITTER: Hickmott JW
PROVIDER: S-EPMC4980111 | biostudies-literature | 2016
REPOSITORIES: biostudies-literature
Hickmott Jack W JW Chen Chih-Yu CY Arenillas David J DJ Korecki Andrea J AJ Lam Siu Ling SL Molday Laurie L LL Bonaguro Russell J RJ Zhou Michelle M Chou Alice Y AY Mathelier Anthony A Boye Sanford L SL Hauswirth William W WW Molday Robert S RS Wasserman Wyeth W WW Simpson Elizabeth M EM
Molecular therapy. Methods & clinical development 20160810
Current gene therapies predominantly use small, strong, and readily available ubiquitous promoters. However, as the field matures, the availability of small, cell-specific promoters would be greatly beneficial. Here we design seven small promoters from the human paired box 6 (PAX6) gene and test them in the adult mouse retina using recombinant adeno-associated virus. We chose the retina due to previous successes in gene therapy for blindness, and the PAX6 gene since it is: well studied; known to ...[more]