Gene Editing Technique Improves Vision in Rats with Inherited Blindness

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Data Show the New "CRISPR/ Cas9" System Potentially Can Be Used to Prevent Retinal Damage in a Type of Retinitis Pigmentosa, One of the Most Common Inherited Disorders of the Retina

January 7, 2016

Blindness Gene Therapy Genetics Rare Diseases Retinitis Pigmentosa

Translational Research

Grantee

A new technique that has the potential to treat inherited diseases by removing genetic defects has been shown for the first time to hinder retinitis pigmentosa, a degenerative eye disease with no known cure that can lead to blindness. A research team at the Cedars-Sinai Board of Governors Regenerative Medicine Institute used a technique known as CRISPR/Cas9 to remove a genetic mutation that causes the blindness disease. The study was published in the journal Molecular Therapy.