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Generation and Genetic Correction of USH2A c.2299delG Mutation in Patient-Derived Induced Pluripotent Stem Cells

Usher syndrome (USH) is the leading cause of inherited combined hearing and vision loss. As an autosomal recessive trait, it affects 15,000 people in the United States alone and is responsible for ~21% of inherited blindness and 3 to 6% of early childhood deafness. Approximately 2/3 of the patients...

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Detalles Bibliográficos
Autores principales: Liu, Xuezhong, Lillywhite, Justin, Zhu, Wenliang, Huang, Zaohua, Clark, Anna M, Gosstola, Nicholas, Maguire, Colin T., Dykxhoorn, Derek, Chen, Zheng-Yi, Yang, Jun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8227183/
https://www.ncbi.nlm.nih.gov/pubmed/34070435
http://dx.doi.org/10.3390/genes12060805