Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene is an early onset and severe cause of blindness. Successful proof-of-concept studies in a canine model have recently shown that development of a corrective gene therapy for RPGR-XLRP may now be an attainable goal. In preparati...

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Vydáno v:Mol Ther
Hlavní autoři: Beltran, William A., Cideciyan, Artur V., Boye, Shannon E., Ye, Guo-Jie, Iwabe, Simone, Dufour, Valerie L., Marinho, Luis Felipe, Swider, Malgorzata, Kosyk, Mychajlo S., Sha, Jin, Boye, Sanford L., Peterson, James J., Witherspoon, C. Douglas, Alexander, John J., Ying, Gui-Shuang, Shearman, Mark S., Chulay, Jeffrey D., Hauswirth, William W., Gamlin, Paul D., Jacobson, Samuel G., Aguirre, Gustavo D.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2017
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5542804/
https://ncbi.nlm.nih.gov/pubmed/28566226
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.05.004
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