Key details Understanding Lesch-Nyhan syndrome: This rare, recessive condition linked to the X chromosome primarily affects boys, and is characterised by a deficiency of the HPRT enzyme, leading to severe motor dysfunction, uric acid overproduction, and compulsive...
A recent study conducted at UMC Utrecht, Netherlands, has shown that mesenchymal stem cells (MSCs) are a safe treatment for brain damage caused by stroke in newborns, and may have a significant positive effect. Perinatal stroke and its consequences Perinatal stroke is...
A groundbreaking therapy based on transplanting mitochondria into blood stem cells has shown encouraging results in an ongoing, phase 2 trial. If successful, the therapy could not only treat rare, incurable diseases, but also potentially help reverse some of the...
Pamela Goldberger was given months to live after a diagnosis of glioblastoma, a highly aggressive brain cancer which has an extremely poor prognosis even with surgery. Now, she is healthy and “living her best life” after taking part in a clinical trial for a novel...
A team of scientists at UCLA, United States, has shown it’s possible to reprogram haematopoietic stem cells to generate a regular supply of cancer-killing T cells. This novel approach, tested in a first-of-its-kind clinical trial the results of which were published in...
The first in-human trial of a stem cell therapy to treat hearing loss is due to begin later this year in the UK, after receiving MHRA approval. The trial will test whether the treatment is safe, as well as evaluate its effects on auditory function.[1] What is hearing...
