Picture CRISPR-Cas9, a gene editing technology, as a GPS-guided scalpel: gRNA directs the Cas9 enzyme, a protein that cuts ...

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

The deletion of two cancer genes, CHD1 and MAP3K7, improves how well tumors respond to cancer immunotherapy and could be used ...

Abu Dhabi successfully completes its first gene therapy for genetic blood disorders, revolutionizing treatment approaches for sickle cell disease and beta thalassemia. Discover the implications of ...

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

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in ...

Innovative research into the gene-editing tool targets influenza’s ability to replicate—stopping it in its tracks.

Gene-editing kicked off 2024 with a bang in the medical sector but closed the year with a whimper. It started with the FDA approval for the first CRSPR-based gene therapy treatment for sickle cell ...

A new study in Nature Communications could influence how gene therapies are designed for many genetic diseases. Pictured: Gokul Ramadoss, the first author, is a PhD candidate in Bruce Conklin's lab at ...