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

“Through CRISPR RNA optimization, we achieved around 59% editing at the TTR locus in our in vitro experiments. In the mouse ...

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations associated with the strategy, including off-target DNA editing. A group of ...

CRISPR gene editing has transitioned from a laboratory curiosity to a cornerstone of modern biotechnology, revolutionizing our approach to genetic diseases, including Charcot-Marie-Tooth (CMT) disease ...

In a new study, the researchers have shown that the CRISPR–Cas3 system can induce reliable, extensive deletions of the transthyretin (TTR) gene and reduce production of TTR protein without any ...