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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Veröffentlicht in:Proc Natl Acad Sci U S A
Hauptverfasser: 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.
Format: Artigo
Sprache:Inglês
Veröffentlicht: National Academy of Sciences 2018
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PNAS Plus
Online Zugang: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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