CRISPR Medicine News, 21 August 2020 | Safe CRISPR gene therapy for hereditary blindness - CRISPR Medicine

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Curing hereditary blindness with CRISPR-Cas without cutting the DNA

Inherited blindness can be treated with a safe gene therapy approach using activation of functionally equivalent genes.
In a proof of principle study, researchers show how 'dead' dCas9 fused to transcriptional activators can 'wake' silent genes to alleviate hereditary blindness in a mouse model of retinitis pigmentosa.
The therapeutic strategy offers safer gene therapy without cutting the DNA and risking indel-type off-target effects, as is a major concern with the classical CRISPR-Cas system.
Read our story and interview with lead researcher Elvir Becirovic here.

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