Article
Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 2 Dec 2020
Lentz Jennifer J, Pan Bifeng, Ponnath Abhilash, Tran Christopher M, Nist-Lund Carl, Galvin Alice, Goldberg Hannah, Robillard Katelyn N, Jodelka Francine M, Farris Hamilton E, Huang Jun, Chen Tianwen, Zhu Hong, Zhou Wu, Rigo Frank, Hastings Michelle L, Géléoc Gwenaëlle S G
Abstract excerpt
Usher syndrome is a syndromic form of hereditary hearing impairment that includes sensorineural hearing loss and delayed-onset retinitis pigmentosa (RP). Type 1 Usher syndrome (USH1) is characterized by congenital profound sensorineural hearing impairment and vestibular areflexia, with adolescent-onset RP. Systemic treatment with antisense oligonucleotides (ASOs) targeting the human USH1C c.216G>A splicing...
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