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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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Dades bibliogràfiques
Publicat a:Genes (Basel)
Autors principals: Liu, Xuezhong, Lillywhite, Justin, Zhu, Wenliang, Huang, Zaohua, Clark, Anna M, Gosstola, Nicholas, Maguire, Colin T., Dykxhoorn, Derek, Chen, Zheng-Yi, Yang, Jun
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2021
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8227183/
https://ncbi.nlm.nih.gov/pubmed/34070435
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/genes12060805
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