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Article

CRISPR Interference-Potential Application in Retinal Disease.

International journal of molecular sciences - 27 Mar 2020

Peddle Caroline F, Fry Lewis E, McClements Michelle E, MacLaren Robert E

Abstract excerpt

The treatment of dominantly inherited retinal diseases requires silencing of the pathogenic allele. RNA interference to suppress gene expression suffers from wide-spread off-target effects, while CRISPR-mediated gene disruption creates permanent changes in the genome. CRISPR interference uses a catalytically inactive 'dead' Cas9 directed by a guide RNA to block transcription of chosen genes without disrupting the...

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Identifiers

PMID: 32230903
DOI: 10.3390/ijms21072329
PMCID: PMC7177328

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