gene therapy

Given the rogue nature of one scientist, should we expect "designer babies" to follow?
To underscore how important the battle for its eradication still is, misperceptions are clarified and key aspects of the inherited illness are addressed here.
FDA-approved gene therapies is atop of the list of exciting health and science advances of 2017. Following in the wake of two cancer therapies approved earlier this year, a third therapy – this time for vision – received a thumbs-up just before the arrival of 2018.   
Scientists report they have successfully treated hemophilia B by giving sufferers gene therapy. When they infuse a gene for the correct blood-clotting factor, it's taken up by the patient who then can produce it on their own. So they no longer have to inject clotting factor to avoid potentially crippling or fatal consequences.          
The combination of a dying boy, a scientific problem that seemed solvable, the right tools and good hands of motivated scientists have created the most amazing science story of the year. Here's how scientists successfully created new skin during this life-saving procedure. 
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. 
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