Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice

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-on...

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Publicado no:Mol Ther
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7704764/
https://ncbi.nlm.nih.gov/pubmed/32818431
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.08.002
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