The Metachromatic Leukodystrophy Initiative (MLDi) critically responds to the findings of Zhang et al. on lentivirus-modified ...

4, 2025 — Research has shown that a potential new targeted therapy ... blocking cancer gene activity, ... Nr1h2: A Key Transcription Factor Unlocks New Potential in Stem Cell-Based Embryo ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Around 1,700 people could be eligible for the one-time treatment, which is seen as a less risky alternative to a donor stem ...

A new gene editing treatment has been approved for use in the NHS, which will help treat sickle cell disease patients ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE ...

The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by ...

A cutting-edge gene therapy has been approved for NHS use for some patients ... It works by editing the faulty gene in a ...

Modified stem cells are then transfused back into the ... There is also a risk of the transplant being rejected. The gene therapy will be available in specialist centres in London, Manchester ...

A GROUNDBREAKING genetic treatment with the potential to cure sickle cell disease will be rolled out on the NHS. The one-off ...