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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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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 |
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