CRISPR-Cas3 genome-editing system holds therapeutic potential
Phys.org • • science
Key Points:
- Transthyretin amyloidosis (ATTR) is a progressive genetic disorder caused by mutations in the TTR gene, leading to harmful amyloid deposits primarily affecting the heart and nerves, with current RNA interference treatments requiring long-term use but not offering a cure.
- Researchers at the University of Tokyo have explored the CRISPR–Cas3 gene-editing system as a novel approach to permanently reduce TTR production by deleting large DNA regions, showing promising results in mouse models with significant gene editing and serum TTR reduction.
- Unlike the widely used CRISPR–Cas9, which makes precise DNA cuts but risks off-target mutations, CRISPR–Cas3 employs a multiprotein complex that degrades DNA over longer stretches, potentially
