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...
Today is World Cord Blood Day! A lot of progress has been made since the very first cord blood transplant was performed in 1988, 37 years ago. Since then, over 60,000 cord blood transplants have been performed worldwide, and donor cord blood stem cells, such as from a...
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...
