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Journal: The Usher Syndrome, a Human Ciliopathy (Pubmed Central) - Jun 28, 2019 Currently, gene-based therapy concepts, such as gene addition, applications of antisense oligonucleotides and TRIDs ("translational readthrough inducing drugs") for the readthrough of nonsense mutations are preclinically evaluated. For USH1B/MYO7A the UshStat gene therapy clinical trial is ongoing.
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Clinical, Journal: Genetic screening of Russian Usher syndrome patients toward selection for gene therapy. (Pubmed Central) - Apr 14, 2019 P=N/A Clinically the prevalence of USH2 is low (39.28%) and the frequency of MYO7A mutations responsible for USH1B is very high (63.63%, N = 7/11) compared to other cohorts. These seven patients carrying MYO7A mutations are preliminarily eligible for the UshStat® gene therapy.
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