Article
Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction.
Nucleic acids research - 21 May 2020
Wang Lingyan, Kempton J Beth, Jiang Han, Jodelka Francine M, Brigande Alev M, Dumont Rachel A, Rigo Frank, Lentz Jennifer J, Hastings Michelle L, Brigande John V
Abstract excerpt
Disabling hearing loss impacts ∼466 million individuals worldwide with 34 million children affected. Gene and pharmacotherapeutic strategies to rescue auditory function in mouse models of human deafness are most effective when administered before hearing onset, after which therapeutic efficacy is significantly diminished or lost. We hypothesize that preemptive correction of a mutation in the fetal inner ear prior...
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