期刊
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
卷 21, 期 7, 页码 -出版社
MDPI
DOI: 10.3390/ijms21072329
关键词
CRISPR interference; CRISPRi; CRISPR; Cas9; RNAi; knock-down; gene therapy; retinal disease; transcriptional repression; dCas9; KRAB
资金
- Royal College of Surgeons of Edinburgh
- Mabel Churn Scholarship
- Rhodes Trust
- North Harbour Club Charitable Trust
- Amar-Franses & Foster-Jenkins Trust
- NIHR Oxford Biomedical Research Centre
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 DNA. It is highly specific and potentially reversible, increasing its safety profile as a therapy. Pre-clinical studies have demonstrated the versatility of CRISPR interference for gene silencing both in vivo and in ex vivo modification of iPSCs for transplantation. Applying CRISPR interference techniques for the treatment of autosomal dominant inherited retinal diseases is promising but there are few in vivo studies to date. This review details how CRISPR interference might be used to treat retinal diseases and addresses potential challenges for clinical translation.
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