Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia
I. Develop an approach for treating patients with homozygous familial hypercholesterolemia using gene therapy with autologous hepatocytes transduced with a normal low-density lipoprotein receptor gene.
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||June 1992|
PROTOCOL OUTLINE: Autologous hepatocytes are obtained from a partial hepatectomy and transduced with a recombinant retroviral vector containing the low-density lipoprotein receptor gene. The transduced hepatocytes are infused via the inferior mesenteric vein 3 days following surgery.
Traditional therapy is discontinued for 4 weeks prior to protocol therapy and may resume 6 weeks after the hepatocyte infusion.
|Study Chair:||James M. Wilson||University of Pennsylvania|