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Autosomal Dominant Retinitis Pigmentosa Due to Class B Rhodopsin Mutations: An Objective Outcome for Future Treatment Trials

Gene therapy for adRP due to RHO mutations was recently shown to prevent photoreceptor death in a canine model of Class B disease. Among translational steps to be taken, one is to determine a method to detect efficacy in a human clinical trial. The relatively slow progression of adRP becomes a diffi...

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Vydáno v:Int J Mol Sci
Hlavní autoři: Sumaroka, Alexander, Cideciyan, Artur V., Charng, Jason, Wu, Vivian, Powers, Christian A., Iyer, Bhavya S., Lisi, Brianna, Swider, Malgorzata, Jacobson, Samuel G.
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2019
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6861901/
https://ncbi.nlm.nih.gov/pubmed/31717845
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20215344
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