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Vassili Valayannopoulos

Regeneron
H指数:55
被引数:1.0万

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DB-OTO Gene Therapy for Inherited Deafness

Valayannopoulos, V.; Bance, M.; Carvalho, D.S.; Greinwald, J.H.; Harvey, S.A.; Ishiyama, A.; Landry, E.C.; Löwenheim, H.; Lustig, L.R.; Manrique, M.; et al. DB-OTO基因疗法用于遗传性耳聋。N. Engl. J. Med. 2025. [Google Scholar] [CrossRef]

2025-10-013
PRE
Functional, sustained recovery of hearing in Otoferlin-deficient mice using DB-OTO, a hair cell-specific AAV based dual vector gene therapy

使用DB-OTO(一种基于毛细胞特异性AAV的双载体基因疗法)实现 otoferlin 缺陷小鼠功能性、持续性的听力恢复

2025-08-250
OAAI
Otof gene transfer in DFNB9 mice carrying human founder non-truncating alleles

携带人类始祖非截断等位基因的DFNB9小鼠中Otof基因的转移

2025-05-160
OAAI
ZBTB11 dysfunction: spectrum of brain abnormalities, biochemical signature and cellular consequences
BRAIN
IF11.7
2022-02-016
OAAI
Neonatal factors related to survival and intellectual and developmental outcome of patients with early-onset urea cycle disorders
2020-06-017
PREAI
Betaine anhydrous in homocystinuria: results from the RoCH registry
2019-03-1414
OAAI
Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants
2016-05-0161
OAAI