retinitis pigmentosa
Inherited Retinal Disease Patients, Therapy Developers May Benefit From New Genetic Testing Program
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To study the benefit of genetic testing data, the Foundation Fighting Blindness offered free clinical testing to 100 members of its My Retina Tracker patient registry.
Look for CRISPR/Cas9 to Treat Eye Diseases First, Scientists Say
For several reasons, scientists, as well as gene editing therapy company Editas, think that the first clinical trials for CRISPR-based cures will be for hereditary eye diseases.
Scientists Fix Retinitis Pigmentosa in Patient-Derived Stem Cells With CRISPR/Cas9
The study hints that iPSCs derived from patients with the hereditary disease could be edited ex vivo and reintroduced to fix damaged retinas.
Genable Technologies, an Irish biopharmaceutical firm, announced last week that it has received orphan drug status from the US Food and Drug Administration for its expressed RNAi-based treatment for retinitis pigmentosa.
Irish biopharmaceutical firm Genable Technologies announced this week that it has acquired an exclusive, worldwide license to Benitec Biopharma's expressed RNAi technology for use in a treatment for the rare ocular disease rhodopsin-linked autosomal dominant retinitis pigmentosa.