In addition to the prolonged pain and suffering endured by sickle cell patients, a Washington University School of Medicine study has concluded that there could be a significant impact on cognitive ...

Kyle Registre's childhood dream of becoming a pilot can finally come true now that he says he's been cured of sickle cell ...

Right now, approximately 20 billion red blood cells are busy traveling through your blood vessels. They are delivering oxygen ...

A groundbreaking sickle cell disease treatment has been approved for use in the UK's National Health Service (NHS). The treatment offers patients with severe forms of the condition hope for a ...

RTCB is required for the splicing of both tRNA and XBP1 mRNA, whereas the DDX1 enzymatic activity is specifically required for tRNA splicing in vivo. Snaebjornsson et al. explore a selective ...

New gene therapies could transform sickle cell treatment forever. A future without pain crises or hospitalizations may finally be within reach.

Sickle cell anaemia is a genetic blood disorder characterised by the production of an abnormal haemoglobin, known as ...

Clinical trials for exa-cel have shown promising results that it has been authorised by the FDA and MHRA for use in beta thalassemia and sickle cell disease. These successes make CRISPR technology, as ...

A "groundbreaking" £1.65 million treatment has been approved for use on the NHS, offering patients with severe sickle cell disease some hope of a cure. It is believed around 1,700 people could be ...

Our independent appraisal committee has approved the use of gene editing therapy exagamglogene autotemcel (exa-cel) for use in the NHS in England, providing a potential cure for some people with ...

A cutting-edge gene therapy has been approved for NHS use for some patients with severe sickle cell disease. Casgevy, also known as exa-cel, was the first treatment to be licensed using gene ...