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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Autores principales: de Leeuw, Charles N, Dyka, Frank M, Boye, Sanford L, Laprise, Stéphanie, Zhou, Michelle, Chou, Alice Y, Borretta, Lisa, McInerny, Simone C, Banks, Kathleen G, Portales-Casamar, Elodie, Swanson, Magdalena I, D’Souza, Cletus A, Boye, Shannon E, Jones, Steven JM, Holt, Robert A, Goldowitz, Daniel, Hauswirth, William W, Wasserman, Wyeth W, Simpson, Elizabeth M
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2014
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3992516/
https://ncbi.nlm.nih.gov/pubmed/24761428
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2013.5
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