Understanding Gene Therapy

What Is Gene Therapy? How Does It Work?

U. S. Food and Drug Administration

U.S.F.D.A. Site

Genetic Therapies

National Institutes of Health

N.I.H. Site

Gene & Cell Therapy Education

American Society of Gene + Cell Therapy

American Society Site

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.

MSUD_Infographic_Gene_Therapy_22Jul19