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hfCas13

Development stage
Preclinical
Lead developer
辉大
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravitreal
01

Overview

hfCas13 (high-fidelity Cas13) is an engineered variant of the CRISPR-associated RNA-guided ribonuclease Cas13, specifically designed to eliminate the "collateral activity" (indiscriminate cleavage of bystander RNA) that is characteristic of wild-type Cas13 enzymes like RfxCas13d. While wild-type Cas13 induces widespread transcriptome destruction upon target recognition, hfCas13 maintains high specificity for its target RNA sequence without triggering non-specific nuclease activity. In research contexts, particularly in oncology and leukemia studies, hfCas13 serves as a precision tool to differentiate between the therapeutic effects of specific gene silencing and the broader cellular impacts of collateral RNA degradation. It is being explored as a platform for high-precision RNA-targeting therapies where off-target transcriptomic disruption must be avoided.

Other names
high-fidelity CRISPR/Cas13high-fidelity Cas13
02

Targets

ssRNA (Target single-stranded RNA)

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