CRISPR gene editing

A groundbreaking CRISPR-based method called SEED/Harvest has been developed by a team at the University of Basel to enable efficient and precise genome modifications in fruit flies without residual damage. This innovation enhances protein research and could significantly advance genetics and medical research. Concurrently, the UK’s National Health Service (NHS) has begun rolling out the CRISPR-based gene therapy Casgevy for patients with transfusion-dependent beta thalassemia, following a positive recommendation from the National Institute for Health and Care Excellence (NICE). Casgevy, which has a list price of £1.65 million, will be available to approximately 460 eligible patients who lack suitable stem cell donors. The therapy involves editing a faulty gene in the patient's bone marrow stem cells to help produce functional hemoglobin, potentially freeing them from lifelong blood transfusions. The introduction of Casgevy marks a significant milestone in gene therapy for blood disorders in the UK.