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
Tag: gene therapy
Advancing gene therapy, debunking AR propaganda.
The promise of curing genetic diseases by replacing damaged genes with healthy ones is slowly becoming a reality. One recent story is the development of therapy in humans to reverse a form of childhood blindness called Leber congenital amaurosis, or LCA. OregonLive reports on the story of Alexe Webb who, soon after birth, was diagnosed … Continue reading Advancing gene therapy, debunking AR propaganda.
The First Decade of the Human Genome: What’s on the Horizon?
To mark the 10th anniversary of the sequencing of the human genome the BBC aired a documentary yesterday evening entitled “Miracle cure: a decade of the human genome” that can be viewed on the BBC iPlayer. It was an enjoyable look at what has been accomplished since the famous announcement at the White House in … Continue reading The First Decade of the Human Genome: What’s on the Horizon?
Animal research: At the forefront of modern medicine
Several reports in the news over the past week have highlighted yet again the importance of animal research to medical advances. The BBC reports that gene therapy has been used successfully to treat a patient with severe β-thalassemia. β-thalassemia is an inherited disorder caused by mutations in the β-globin chain of haemoglobin that lead to … Continue reading Animal research: At the forefront of modern medicine
Breakthrough of the Year (almost!)
As the year draws to a close it’s time to reflect on an exciting year of animal research, and there seems no better place to start than with the top 10 breakthroughs of the year as selected by the prestigious scientific journal Science. Science is of course a general science magazine, and the choices reflect … Continue reading Breakthrough of the Year (almost!)
Gene therapy on the brain
Hot on the heels of last weeks report of the successful use of gene therapy to treat the eye disease Leber’s congenital amaurosis comes a report that scientists lead by Nathalie Cartier and Patrick Aubourg of the French National Institute for Health and Medical Research have combined gene therapy and stem cell medicine to successfully … Continue reading Gene therapy on the brain
Gene therapy for blindness – when dogged determination pays off!
Leber's congenital amaurosis is a progressive disorder that affects about 3,000 Americans, and hundreds of thousands worldwide, and causes a progressive loss of vision that usually results in blindness. The disease, for which there has until now been no effective treatment, is caused by a mutation in the encoding RPE65, an enzyme which is crucial … Continue reading Gene therapy for blindness – when dogged determination pays off!
Blind Dogs Lead Researchers to Treatments
Leber’s congenital amaurosis (LCA) is a form of blindness that affects about 1 in 80,000 people. This inherited disease, in which the retina progressively degenerates, results in severe loss of vision, and frequently patients can only see well enough to count fingers or see bright lights. Unfortunately, many of these patients also experience eye pain from … Continue reading Blind Dogs Lead Researchers to Treatments
Speaking of Research 