Good News Notes: “After a major cardiac event, the heart can’t repair itself, leaving permanent damage that increases the risk of future troubles. Now, scientists at Texas Heart Institute believe they may have found a viable strategy to help the heart regenerate by way of a gene therapy. The gene therapy uses an adeno-associated virus (AAV)…
Tag: gene therapy
Researchers develop breath test for rare inherited disorder
Good News Notes: “Methylmalonic acidemia (MMA) is a rare genomic condition that disrupts the normal metabolism of certain proteins and fats, resulting in a toxic accumulation of methylmalonic acid in the body. This build-up may lead to irreparable kidney impairment, metabolic liver dysfunction, pancreatitis, intellectual disability and eventually, without treatment, death. Researchers at the National…
Scientists find a switch to flip and turn off breast cancer growth and metastasis
Good News Notes: “Researchers at Tulane University School of Medicine identified a gene that causes an aggressive form of breast cancer to rapidly grow. More importantly, they have also discovered a way to “turn it off” and inhibit cancer from occurring. The animal study results have been so compelling that the team is now working…
Gene therapy trial shows promise for curing a type of hemophilia
Good News Notes: “A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate. Patients who received the one-time intravenous therapy continue to have a more than 90 percent decrease in bleeding events two to three years after their initial treatment, researchers reported…
How a Pharmaceutical Company Developed a Breakthrough Treatment for Cystic Fibrosis
Good News Notes: “In June 1989, Dr. Lap-Chee Tsui and Dr. Francis Collins, two geneticists, pinpointed the gene responsible for cystic fibrosis, the cystic fibrosis transmembrane conductance regulator (CFTR) gene.” “In the decade that followed, scientists experimented with gene therapy, a method of replacing defective genes with healthy ones, but to no avail.” “Eventually, in…