Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors

Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors

Critical for human gene therapy is the availability of small promoters tools to drive gene expression in a highly specific and reproducible manner. We tackled this challenge by developing human DNA MiniPromoters (MiniPs) using computational biology and phylogenetic conservation. MiniPs were tested i...

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Bibliographic Details
Main Authors: Charles N de Leeuw, Frank M Dyka, Sanford L Boye, Stéphanie Laprise, Michelle Zhou, Alice Y Chou, Lisa Borretta, Simone C McInerny, Kathleen G Banks, Elodie Portales-Casamar, Magdalena I Swanson, Cletus A D'Souza, Shannon E Boye, Steven JM Jones, Robert A Holt, Daniel Goldowitz, William W Hauswirth, Wyeth W Wasserman, Elizabeth M Simpson
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116300705

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http://www.sciencedirect.com/science/article/pii/S2329050116300705

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