The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending a CRISPR gene editing-based treatment for severe sickle cell disease (SCD). The regulator said ...

In December 2020, the New England Journal of Medicine published a paper titled CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia, marking an exciting new chapter in the treatment of ...

UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

This story is republished from STAT, the health and medicine news site that’s a partner to the Globe. Sign up for STAT’s free Morning Rounds newsletter here. Vertex executives warned that Casgevy, its ...

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with ...

CRISPR-Cas 9 is a gene-editing tool that made it possible to rewrite any organism's genetic code and tackle genetic diseases more effectively. Known as genetic scissors, CRISPR identifies a DNA ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...