The root causes of most forms of Alzheimer's disease largely remain a mystery. Now, researchers have revealed a new piece of ...

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.

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 ...

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 ...

A sickle cell disease “cure” is to be offered to NHS patients for the first time. The £1.65 million gene therapy called exa-cel, also known under the brand name Casgevy, uses a Nobel Prize ...

Exa-cel provides a new hope for people with sickle cell disease to have a cure. Sickle cell disease is caused by a mutation in the HBB gene, which encodes the beta-globin subunit of hemoglobin. This ...

“The background is that sickle cell disease is caused by mutation of one of the proteins that form haemoglobin ... The therapy works by editing the patient’s BCL11A gene to reactivate fetal ...

LONDON, Jan 31 (Reuters) - Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care ...

Sickle-cell anaemia is the name of a specific form of sickle-cell disease in which there is homozygosity for the mutation that causes HbS. Sickle-cell anaemia is also referred to as "HbSS", "SS ...

Individuals with SCT have one copy of the gene associated with sickle cell disease (SCD). SCD is a blood disorder characterized by misshapen blood cells that can cause blockages, leading to ...

Sickle cell anaemia, a hereditary condition that primarily affects African and Mediterranean populations, continues to pose significant health challenges. In Uganda, where the disease is prevalent ...