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Optimized AAV Vectors for TMC1 Gene Therapy in a Humanized Mouse Model of DFNB7/11

Gene therapy for genetic hearing loss is an emerging therapeutic modality for hearing restoration. However, the approach has not yet been translated into clinical application. To further develop inner-ear gene therapy, we engineered a novel mouse model bearing a human mutation in the transmembrane c...

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Principais autores: Irina Marcovich, Nicholas K. Baer, Olga Shubina-Oleinik, Rachel Eclov, Clayton W. Beard, Jeffrey R. Holt
Format: Artigo
Sprog:Inglês
Udgivet: MDPI AG 2022-06-01
Serier:Biomolecules
Fag:
inner ear
hair cell
cochlea
vestibular
utricle
saccule
Online adgang:https://www.mdpi.com/2218-273X/12/7/914
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