Understanding Gene Therapy

              What Is Gene Therapy? How Does It Work?

 

                U. S. Food and Drug Administration

        Learn More

 

Genetic Therapies

National Institutes of Health 

Learn More

 

 

Gene & Cell Therapy Education

American Society of Gene + Cell Therapy

Learn More

 

Infographic On MSUD Gene Therapy

Experimental, molecular therapies, including delivery of messenger RNA (mRNA) and AAV gene therapy strive to overcome the genetic mutation(s) causing MSUD in different ways. To download this infographic, click here.