Opinion | A New Sickle Cell Treatment Will Change Lives — but How Many?
A new treatment for sickle cell disease will change lives — but how many?
Stay updated with breaking news from Crispr. Get real-time updates on events, politics, business, and more. Visit us for reliable news and exclusive interviews.
A new treatment for sickle cell disease will change lives — but how many?
An advisory committee is meeting to discuss the potential approval of exagamglogene autotemcel, a CRISPR-based gene editing therapy candidate for sickle cell disease.
The treatment from the company Vertex would be the first medicine to use the gene editing tool CRISPR.
In a Perspective, Stephen Tang and Samuel Sternberg discuss retroelement-based gene editing as a safer alternative to CRISPR-Cas approaches.
Severe Combined Immunodeficiencies (SCIDs) are a group of debilitating primary immunodeficiency disorders, primarily caused by genetic mutations that disrupt T-cell development. SCID can also affect B-cell and natural killer cell function and counts.