The new therapy, a collaboration between researchers at the ... so scientists overcame this hurdle by using a protein fusion ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...

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 ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Figure 1: Diagram of the anagen hair follicle ... a characteristic which could be exploited by in vivo and ex vivo gene therapy to produce molecules other than those involved directly in hair ...

Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders. Now it’s on the cusp of its first regulatory approval. Each year ...

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.

“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 ...