Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector

Inherited retinal degenerations are caused by mutations in >250 genes that affect photoreceptor cells or the retinal pigment epithelium and result in vision loss. For autosomal recessive and X-linked retinal degenerations, significant progress has been achieved in the field of gene therapy as evi...

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Detalhes bibliográficos
Publicado no:Proc Natl Acad Sci U S A
Main Authors: Cideciyan, Artur V., Sudharsan, Raghavi, Dufour, Valérie L., Massengill, Michael T., Iwabe, Simone, Swider, Malgorzata, Lisi, Brianna, Sumaroka, Alexander, Marinho, Luis Felipe, Appelbaum, Tatyana, Rossmiller, Brian, Hauswirth, William W., Jacobson, Samuel G., Lewin, Alfred S., Aguirre, Gustavo D., Beltran, William A.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2018
Assuntos:
PNAS Plus
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6130384/
https://ncbi.nlm.nih.gov/pubmed/30127005
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1805055115
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