Joseph BURNS,Kathryn ELLIS,Adam PALERMO,Martin SCHWANDER,Jonathon WHITTON
申请号：
US16395999
公开号：
US20200155705A1
申请日:
2019.04.26
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The disclosure features compositions and methods for the treatment of sensorineural hearing loss and auditory neuropathy, particularly forms of the disease that are associated with mutations in otoferlin (OTOF), by way of OTOF gene therapy. The disclosure provides a variety of compositions that include a first nucleic acid vector that contains a polynucleotide encoding an N-terminal portion of an OTOF protein and a second nucleic acid vector that contains a polynucleotide encoding a C-terminal portion of an OTOF protein. These vectors can be used to increase the expression of OTOF in a subject, such as a human subject suffering from sensorineural hearing loss.
