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PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina

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 the...

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Autori principali: Jack W Hickmott, Chih-yu Chen, David J Arenillas, Andrea J Korecki, Siu Ling Lam, Laurie L Molday, Russell J Bonaguro, Michelle Zhou, Alice Y Chou, Anthony Mathelier, Sanford L Boye, William W Hauswirth, Robert S Molday, Wyeth W Wasserman, Elizabeth M Simpson
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2016-01-01
Serie:Molecular Therapy: Methods & Clinical Development
Accesso online:http://www.sciencedirect.com/science/article/pii/S2329050117300232
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