期刊
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
卷 22, 期 8, 页码 -出版社
MDPI
DOI: 10.3390/ijms22083910
关键词
syndromic hearing loss; retinitis pigmentosa; CRISPR; antisense oligonucleotides
资金
- NIH [R01DC005575, R01DC012115]
Hearing loss is a common sensory disorder affecting approximately 466 million people worldwide, with a significant portion being genetic. Usher syndrome is a syndromic form of genetic hearing loss that is often accompanied by retinitis pigmentosa and vestibular dysfunction. Currently, cochlear implantation or hearing aids are the main treatment options for Usher-related hearing loss.
Hearing loss is the most common sensory disorder with similar to 466 million people worldwide affected, representing about 5% of the population. A substantial portion of hearing loss is genetic. Hearing loss can either be non-syndromic, if hearing loss is the only clinical manifestation, or syndromic, if the hearing loss is accompanied by a collage of other clinical manifestations. Usher syndrome is a syndromic form of genetic hearing loss that is accompanied by impaired vision associated with retinitis pigmentosa and, in many cases, vestibular dysfunction. It is the most common cause of deaf-blindness. Currently cochlear implantation or hearing aids are the only treatments for Usher-related hearing loss. However, gene therapy has shown promise in treating Usher-related retinitis pigmentosa. Here we review how the etiologies of Usher-related hearing loss make it a good candidate for gene therapy and discuss how various forms of gene therapy could be applied to Usher-related hearing loss.
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