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Gene therapy in animal models of autosomal dominant retinitis pigmentosa

Gene therapy for dominantly inherited genetic disease is more difficult than gene-based therapy for recessive disorders, which can be treated with gene supplementation. Treatment of dominant disease may require gene supplementation partnered with suppression of the expression of the mutant gene eith...

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Detalhes bibliográficos
Main Authors: Rossmiller, Brian, Mao, Haoyu, Lewin, Alfred S.
Formato: Artigo
Idioma:Inglês
Publicado em: Molecular Vision 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3472929/
https://ncbi.nlm.nih.gov/pubmed/23077406
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