adenovirus

Successful Gene Therapy for Rare Hemophilia

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.

Progress on Gene Modification Rx for Muscular Dystrophy

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.