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PKHD1L1 is required for stereocilia bundle maintenance, durable hearing function and resilience to noise exposure.
Strelkova OS, Osgood RT, Tian CJ, Zhang X, Hale E, De-la-Torre P, Hathaway DM, Indzhykulian AA. Strelkova OS, et al. Among authors: hathaway dm. bioRxiv [Preprint]. 2024 Mar 27:2024.02.29.582786. doi: 10.1101/2024.02.29.582786. bioRxiv. 2024. Update in: Commun Biol. 2024 Nov 1;7(1):1423. doi: 10.1038/s42003-024-07121-5 PMID: 38496629 Free PMC article. Updated. Preprint.
Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F.
Ivanchenko MV, Hathaway DM, Klein AJ, Pan B, Strelkova O, De-la-Torre P, Wu X, Peters CW, Mulhall EM, Booth KT, Goldstein C, Brower J, Sotomayor M, Indzhykulian AA, Corey DP. Ivanchenko MV, et al. Among authors: hathaway dm. Nat Commun. 2023 Apr 26;14(1):2400. doi: 10.1038/s41467-023-38038-y. Nat Commun. 2023. PMID: 37100771 Free PMC article.
PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F.
Ivanchenko MV, Hathaway DM, Mulhall EM, Booth KT, Wang M, Peters CW, Klein AJ, Chen X, Li Y, György B, Corey DP. Ivanchenko MV, et al. Among authors: hathaway dm. bioRxiv [Preprint]. 2023 Nov 13:2023.11.09.566447. doi: 10.1101/2023.11.09.566447. bioRxiv. 2023. Update in: J Clin Invest. 2024 Oct 23;134(23):e177700. doi: 10.1172/JCI177700 PMID: 38014037 Free PMC article. Updated. Preprint.
AAV-S: A versatile capsid variant for transduction of mouse and primate inner ear.
Ivanchenko MV, Hanlon KS, Hathaway DM, Klein AJ, Peters CW, Li Y, Tamvakologos PI, Nammour J, Maguire CA, Corey DP. Ivanchenko MV, et al. Among authors: hathaway dm. Mol Ther Methods Clin Dev. 2021 Mar 29;21:382-398. doi: 10.1016/j.omtm.2021.03.019. eCollection 2021 Jun 11. Mol Ther Methods Clin Dev. 2021. PMID: 33869656 Free PMC article.