CRISPR–Cas3 genome-editing system holds therapeutic potential

Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.
Seeking Alpha

Crispr And Intellia: Advancing In Vivo Gene Editing As 2025 Results Strengthen Investment Outlook

Crispr Therapeutics (CRSP) is downgraded to "Hold" as current valuation already reflects optimism for CTX310 Phase I results and pipeline progress. Intellia Therapeutics (NTLA) maintains a "Buy" ...
NPR

A promising genetic treatment tailor-made for a baby born with a rare disorder

For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby ...
The New England Journal of Medicine

Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3

Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...