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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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Dyka, Frank M., Boye, Sanford L., Chiodo, Vince A., Hauswirth, William W., Boye, Shannon E.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Mary Ann Liebert, Inc. 2014
Gaiak:
Sarrera elektronikoa: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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