Pigmentosa

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Studies in non-human primates demonstrated nearly 100% gene editing and knockout of endogenous RHO gene and more than 30% replacement protein levels using a dual vector AAV approach…

Pune, July 04, 2022 (GLOBE NEWSWIRE) -- Retinitis Pigmentosa Treatment And Management Market by Vendor Assessment, Technology Assessment, Partner & Customer Ecosystem, type/solution,…

• The primary outcome of the MGT009 study is safety, and botaretigene sparoparvovec treatment was found to be generally safe and well-tolerated • Significant improvements were…

Retinitis pigmentosa, a degenerative genetic disease of the eye, is characterized by progressive vision loss, usually leading to blindness. In some patients, structural defects in the…

Studies in non-human primates demonstrated nearly 100% gene editing knockout of endogenous RHO gene and more than 30% replacement protein levels Treated eyes showed morphological and…

Researchers from the University of California, Irvine have discovered that the absence of Adiponectin receptor 1 protein (AdipoR1), one of the principal enzymes regulating ceramide…

4D-125 was well tolerated in all patients treated to-date (n=8), with no dose-limiting toxicities, no serious adverse events and no chronic inflammationClinical activity observed through…