Cas13d-MT

Cas13d-MT is an engineered CRISPR-Cas13d system specifically designed for the targeted degradation of mitochondrial RNA (mtRNA). It consists of the RfxCas13d protein fused with a mitochondrial targeting signal (MTS), which enables the complex to bypass the mitochondrial membrane and localize within the mitochondrial matrix. Once inside, the Cas13d-MT, guided by a specific CRISPR RNA (crRNA), identifies and cleaves complementary mutant mtRNA transcripts. This technology is primarily being developed to treat mitochondrial diseases—such as Leber's Hereditary Optic Neuropathy (LHON)—by selectively reducing the burden of pathogenic RNA mutations. By shifting heteroplasmy levels and eliminating toxic RNA species, Cas13d-MT aims to restore mitochondrial function in cells affected by mitochondrial genome mutations that are otherwise inaccessible to standard DNA-targeting CRISPR systems.

Other names

Mitochondria-targeted Cas13dmito-Cas13dmito-Cas-13dmito-Cas 13d