Article
Gene supplementation with precise transgene expression rescues hearing loss in a mouse model with an Mpzl2 East Asian founder variant.
Molecular therapy : the journal of the American Society of Gene Therapy - 7 Jan 2026
Jang Seung Hyun, Song Hyeong Gi, Joo Sun Young, Kim Jung Ah, Kim Se Jin, Choi Jae Young, Jung Jinsei, Gee Heon Yung
Abstract excerpt
Hearing loss is the most common sensory organ disorder, with genetic factors contributing substantially to the disease. Among the 87 genes responsible for autosomal recessive nonsyndromic hearing loss, mutations in MPZL2 have been frequently linked to mild-to-moderate autosomal recessive hearing loss (DFNB111). Here, we present multiple families whose hearing loss arose from biallelic mutations in the MPZL2 gene...
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