USH2A Gene Editing Using the CRISPR System
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چکیده
منابع مشابه
USH2A Gene Editing Using the CRISPR System
Usher syndrome (USH) is a rare autosomal recessive disease and the most common inherited form of combined visual and hearing impairment. Up to 13 genes are associated with this disorder, with USH2A being the most prevalent, due partially to the recurrence rate of the c.2299delG mutation. Excluding hearing aids or cochlear implants for hearing impairment, there are no medical solutions available...
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ژورنال
عنوان ژورنال: Molecular Therapy - Nucleic Acids
سال: 2017
ISSN: 2162-2531
DOI: 10.1016/j.omtn.2017.08.003