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
Tag: mouse
Animal research unleashes the power of human embryonic stem cells
For more than a decade now embryonic stem cell research has been one of the most high profile – and indeed controversial - areas of medical science, and it is an emerging field that owes a lot to animal studies performed by pioneers like Gail Martin of UCSF. Recently the field has begun to live … Continue reading Animal research unleashes the power of human embryonic stem cells
Mice and macaques pave the way for effective HIV vaccines
There is encouraging news this week on the prospects for an effective vaccine against HIV. A research team led by Professor Mariano Esteban at the Spanish Superior Scientific Research Council (CSIC) have announced that the vaccine MVA-B elicited a persistent immune response against HIV in 85% of volunteers in a phase 1 clinical trial. MVA-B … Continue reading Mice and macaques pave the way for effective HIV vaccines
Mice show the way to improved stem cell therapy for heart attacks
When the results of clinical trials do not live up to expectations from pre-clinical studies in animals it can be all too easy to ascribe the divergence to species differences, however scientists are increasingly aware that in many, even most cases, the problem may not be species differences but rather differences in the design of studies in … Continue reading Mice show the way to improved stem cell therapy for heart attacks
A breakthrough against Chronic Lymphocytic Leukemia…thank the mice!
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!
Honoring a fallen hero in the struggle against Cystic Fibrosis and AR extremism
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
Promising clinical trial result for exon skipping in Duchenne Muscular Dystrophy
Last Monday the Muscular Dystrophy Campaign announced that the antisense oligonucleotide drug AVI-4658 had performed well in its second clinical trial in 19 boys with Duchenne Muscular Dystrophy (DMD). DMD is one of the most common hereditary disorders affecting the skeletomuscular system, and which results in progressive muscle degeneration, difficulty in walking and breathing, and … Continue reading Promising clinical trial result for exon skipping in Duchenne Muscular Dystrophy
Lighting the Way to New Treatments
A variety of diseases in humans happen when proteins with important cellular functions are lacking or are produced in abnormally low amounts. One example is type-2 diabetes mellitus which is caused by a complex set of problems involving the use of sugars (mostly, glucose) as an energy source. After eating, sugars in food are taken … Continue reading Lighting the Way to New Treatments
Speaking of Research 