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Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A

Usher syndrome 1B (USH1B) is a severe, autosomal recessive, deaf–blind disorder caused by mutations in myosin 7A (MYO7A). Patients are born profoundly deaf and exhibit progressive loss of vision starting in their first decade. MYO7A is expressed in human photoreceptors and retinal pigment epithelium...

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Detalhes bibliográficos
Main Authors: Dyka, Frank M., Boye, Sanford L., Chiodo, Vince A., Hauswirth, William W., Boye, Shannon E.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2014
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3991978/
https://ncbi.nlm.nih.gov/pubmed/24568220
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2013.212
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