CRISPR and Vertex's two phase 1/2 studies in thalassaemia and SCD – called CLIMB-111 and CLIMB-211, respectively – have now recruited 19 patients. They are due to enrol 45 apiece, and are ...

The Department of Health – Abu Dhabi (DoH), the regulator of the healthcare sector in the emirate, has announced the introduction of CASGEVY, the first CRISPR/Cas9 gene-editing therapy in the UAE.

Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...

Vertex Pharma's £1.65 million ($2.05 million) gene-editing therapy for sickle cell disease (SCD), Casgevy ... and with a confidential discount. The CRISPR-based therapy will be an option for ...

Medscape Medical News, December 19, 2023 ASH 2023 Sickle Cell CRISPR Therapy May Offer 'Functional Cure' Patients with severe sickle cell disease could soon be freed from vaso-occlusive crises ...

Understanding the differences in the mutations that ... FLIpping the Switch: Boosting Stem Cell Numbers for Therapies Mar. 10, 2025 — A single molecular switch is essential for blood stem cells ...

Cell biology is the discipline of biological sciences that studies the structure, physiology, growth, reproduction and death of cells. Research in cell biology uses microscopic and molecular tools ...

CRISPR-Cas systems are prokaryotic immune systems that confer resistance to foreign genetic elements such as plasmids and phages. CRISPR-Cas systems have been exploited for targeted genome editing ...

One such strategy involves the integration of orthogonal experimental designs with advanced genomic tools like CRISPR libraries and single-cell sequencing. Orthogonal experiments streamline ...

Scientists have found a potential new gene variant target to treat sickle cell disease, an inherited blood disorder that affects about 300,000 people globally each year, with limited treatment options ...

Expanding CRISPR's Potential Since its discovery, CRISPR (“Clustered Regularly Interspaced Short Palindromic Repeats”), which ...