Scientists Cure Hemophiliacs

Scientist use gene therapy to cure hemophilia…

Scientists say they have successfully cured four patients with Hemophilia – a disease the causes defective blood clotting which leads to excessive bleeding that can kill.

After years of preclinical trials, which including curing hemophiliac mice, scientist finally started trials on humans using gene therapy.

The patients were injected with a specially built virus containing a working version of the gene responsible for blood clotting. The virus implants the gene into liver cells where they begin to produce the chemical need for clotting.

Red and White Blood Cells

After 6 months of injections to maintain elevated levels of the working gene, four of them were able to stop receiving injections altogether.

The researchers are now monitoring the patients for any signs of liver cancer caused by the virus, a known risk of gene therapy, but so far there have been no complications.

The next stage will be the trial of 20 patients to assess what dosage of the virus is necessary to get enough liver cells making clotting factor so that all patients can stop receiving injections.

The study was published in the New England Journal of Medicine.

Stem Cells Safe Procedure To Treat Blindness

The world’s first clinical trials using embryonic stem cells to treat blindness suggest the technique is safe…

The world’s first human trial using stem cells to treat blindness, suggests that the procedure is safe.

US-based Advanced Cell Technology together with the Jules Stein Eye Institute at the University of California, Los Angeles, have announced their first results, which showed the embryonic stems can be successful attached to deteriorated retina, without showing signs of rejection.

Trials using the same technique have now begun at London’s Moorfields Eye Hospital.

The trials involved two patients; one elderly woman in her 70s with dry age-related macular degeneration, and another female patient in her 50s with Stargardt’s disease. Both had very poor vision and were registered blind.

The team injected a mixture, containing 50,000 retinal cells, into the eyes of the patients. The cells then attached to the retinal membrane and grow to replicate the function of other healthy cells surrounding it.

Four months after surgery, both patients were doing well, the team reported.

While the study was conducted to test if the procedure is safe, and not to test if it actually works, the patients did report improved vision following the operation.

If proved safe, the treatment should be able to treat patients with currently incurable blindness, however since using embryonic stem cells is a highly controversial topic, the technique will face great opposition, even if it’s deemed effective.

The team also notes that much more research needs to be conducted, and that it will be years before the treatment can be proven. Nevertheless, the results are certainly a step in the right direction for those who advocate stem cell therapy.