ACSH's Director of Medicine, Dr. Jamie Wells, traveled to Washington, DC to tape at Al Jazeera for a live television program. It engaged a global discussion on sickle cell anemia, its perils and the advances in gene therapy that are showing great promise for this genetic disease.
gene therapy
A number of recent headlines imply that a new case study in the New England Journal of Medicine proves that gene therapy has cured sickle cell disease — a genetic disorder that causes tremendous pain, suffering and diminished life expectancy. Let's unpack the significance of the researchers’ findings.
A new study reveals how researchers were able to use a single gene therapy injection in dogs to successfully correct a rare type of hemophilia. Factor VII deficiency is an autosomal recessive bleeding disorder that affects about one in 500,000 people.
Three independently-working groups have developed an effective, albeit temporary, therapy for Duchenne Muscular Dystrophy to delete the defective gene and replace it with a functional gene. Although these succeeded in mice, human treatment will not be far behind.
A study of elephants finds these massive animals get cancer at a far lower rate than humans. Researchers also found the reason: they have an astronomical number of copies of a vital gene. The news is interesting, but is unlikely to do more for improving human health.
It has been 25 years since the first clinical trial of gene therapy was conducted, but one still has not been approved in the U.S. However, following two new studies showing that researchers are closing in on therapies for a common brain cancer and a rare eye disease, hope for approval is on the rise.
Scientists and doctors alike have considered gene therapy a potential panacea since it was first postulated on in the 1970s. If harnessed correctly, it is theorized gene therapy could provide real cures for an
A small pilot study of gene therapy for Parkinson s disease showed improvement in severe motor symptoms. There were no side effects of significance, and all three doses had beneficial effect.