Gene therapy using LDL receptor gene data

Researchers reported in Nature Genetics the clinical results of gene therapy in a patient with familial hypercholesterolemia due to a defective gene for the LDL receptor.

The patient's liver was resected and the hepatocytes grown in the lab in the presence of a retrovirus containing the LDL receptor gene. On infusion back into the vein leading to the liver, the cells appear to have engrafted. ...