The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

How does gene therapy work? The currently licensed gene therapies for hemophilia A and B deliver genes via adeno-associated viruses. “You’re basically programming the body to make the missing ...

Current gene therapy treatments work to control and minimize the symptoms, which can be drastic. The estimated lifetime medical costs for SCD are around $1.2 million. According to the Muscular ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in gene therapy using adeno-associated virus vectors (AAV), specifically ...

D.C., for his stem cell collection — the first major step of the revolutionary sickle cell gene therapy process. “But that also comes with the skepticism of: Will it really work?” ...

The scientific advancements of the past 30 years have transformed our understanding of the potential of gene therapy and how it ... with rare diseases still do not have access to the right care ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first ...

President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program ...