Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

Gene Therapy Loses Luster as Investors Eye Quicker Returns From Weight-Loss Drugs By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell ...

Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw ...

“This is the beginning of a new era of medicine,” said Dr. Kiran Musunuru, a gene therapy researcher at the University of Pennsylvania’s Perelman School of Medicine. He added that the method ...

Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition. For more than 15 years ...

A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine disease (MSUD), suggesting the approach could one day be used to treat ...

Gene therapy targeting maple syrup urine disease (MSUD) has shown potential in preventing newborn death, normalizing growth, and stabilizing biomarkers in animal models, including calves and mice.