Italian scientists announced yesterday that they had successfully treated the deadly genetic disorders metachromatic leukodystrophy and Wiskott-Aldrich syndrome in a small clinical trial, marking and another major landmark in the developing field of gene therapy. As you might expect the breakthrough has received widespread media coverage, including CBS, the BBC and the Italian edition of the Huffington Post. While … Continue reading Italian Gene Therapy Success – Thanks to Animal Research
A couple of days ago the New York times published a heart warming story about a young girl named Emma Whitehead whose acute lymphoblastic leukemia - which had previously defied all therapies - has gone into full remission following treatment with a novel gene therapy that programmed her immune system to target the cancer cells. … Continue reading Defeating Leukemia: A smile that says “Thank the mice”
Mitochondria are fascinating. These tiny organelles that reside within almost all of the cells in our bodies (mature red blood cells being an exception) generate the supply of a molecule called adenosine triphosphate (ATP) which is the principle source of energy that cells, and ultimately ourselves, need to survive. They also have an intriguing evolutionary … Continue reading New gene therapy for mitochondrial diseases a step closer thanks to ONPRC
As gene therapy emerges as one of the hottest areas of medical research, one thing that is striking is how it employs viruses - sometimes very nasty viruses - to deliver the gene to where it is needed in the human body. Yesterday virologist Abbie Smith discussed another excellent example of this on the ERV blog … Continue reading ERV blogs on GMO Herpes vs severe cancer pain
Earlier today the BBC reported that European Stroke Research Network for Hypothermia (EuroHYP) has announced the launch of a major clinical trial – involving 1,500 patients in 15 centers across Europe – to evaluate whether cooling the body by 2 degrees can reduce the risk of death and disability in ischaemic stroke. The trial, known … Continue reading Hypothermia in stroke: EuroHYP moves from rats to man
That was the headline of an editorial in the New England Journal of Medicine (NEJM) which discussed the very promising results of a small clinical trial of gene therapy to treat hemophilia B – also known as Christmas Disease*. Patients with haemophilia B suffer bleeding in the joints and muscles due to deficiency in a … Continue reading Merry Christmas for Patients with Hemophilia B
A challenge that science communicators frequently face when discussing the process whereby a scientific discovery eventually leads to a medical breakthrough is the time that this often takes, indeed by the time that the reports of exciting clinical trial outcomes start to appear in the press the role of the scientists who made the initial … Continue reading A breakthrough against Chronic Lymphocytic Leukemia…thank the mice!
Yesterday I learned some sad news via the Understanding Animal Research blog, that a young woman named Laura Cowell had died, succumbing to cystic fibrosis at the age of just 25. To see a life so full of promise end so prematurely is always sad, but what makes this death so gutting is that Laura … Continue reading Honoring a fallen hero in the struggle against Cystic Fibrosis and AR extremism