Journal
JOURNAL OF CLINICAL INVESTIGATION
Volume 132, Issue 9, Pages -Publisher
AMER SOC CLINICAL INVESTIGATION INC
DOI: 10.1172/JCI158287
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Funding
- NIH [5P30CA013696, R01EY09076, U01EY030580, U54OD020351, R24EY028758, R24EY027285, 5P30EY019007, R01EY018213, R01EY024698, R01EY026682, R21AG050437]
- Schneeweiss Stem Cell Fund, New York State [SDHDOH01-C325-90GG-3450000]
- Foundation Fighting Blindness New York Regional Research Center grant [TA-NMT-0116-0692-COLU]
- Crowley Family Funds
- Rosenbaum Family Foundation
- Alcon Research Institute
- Gebroe Family Foundation
- Research to Prevent Blindness (RPB) Physician-Scientist Award
- RPB, New York, New York, USA
- Curing Retinal Blindness Foundation
- Knights Templar Eye Foundation Career Starter grant
- International Retinal Research Foundation Loris and David Rich Postdoctoral Scholar Award
- New York Stem Cell Foundation-Druckenmiller Fellowship
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This article details recent advancements in CRISPR therapeutics for treating a wide range of autosomal dominant disorders and discusses how these advancements are shaping the future of therapies.
Autosomal dominant disorders present unique challenges, as therapeutics must often distinguish between healthy and diseased alleles while maintaining high efficiency, specificity, and safety. For this task, CRISPR/Cas remains particularly promising. Various CRISPR/Cas systems, like homology-directed repair, base editors, and prime editors, have been demonstrated to selectively edit mutant alleles either by incorporating these mutations into sgRNA sequences (near the protospacer-adjacent motif [???near the PAM???]) or by targeting a novel PAM generated by the mutation (???in the PAM???). However, these probability-based designs are not always assured, necessitating generalized, mutation-agnostic strategies like ablate-and-replace and single-nucleotide polymorphism editing. Here, we detail recent advancements in CRISPR therapeutics to treat a wide range of autosomal dominant disorders and discuss how they are altering the landscape for future therapies.
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