First results of long-term follow-up of children in Russia after gene therapy for hereditary retinal dystrophies associated with biallelic mutations in the RPE65 gene

Purpose: to evaluate the results of gene therapy by the recombinant adeno-associated viral vector voretigene neparvovec (VN) in children with follow-up periods of 1, 3, 6, and 12 months.Material and methods. The study included 6 children (12 eyes) aged 5 to 15 with a confirmed biallelic mutation in...

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Principais autores:	V. V. Neroev, L. A. Katargina, M. P. Kharlampidi, L. V. Kogoleva, I. V. Zolnikova, P. A. Ilyukhin, E. V. Denisova, S. V. Milash, N. A. Osipova, S. I. Kutsev, A. V. Polyakov, R. A. Zinchenko, V. V. Kadyshev, Yu. A. Bobrovskaya
Formato:	Artigo
Idioma:	Russo
Publicado em:	Real Time Ltd 2023-12-01
Colecção:	Российский офтальмологический журнал
Assuntos:	inherited retinal dystrophies gene therapy rpe65 voretigen neparvovec luxturna leber congenital amaurosis retinitis pigmentosa genetics, ophthalmology
Acesso em linha:	https://roj.igb.ru/jour/article/view/1356
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