A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia

@article{Grossman1995APS,
  title={A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia},
  author={M. Grossman and D. Rader and D. Muller and D. Kolansky and K. Kozarsky and B. Clark and E. Stein and P. Lupien and H. Brewer and S. Raper and J. Wilson},
  journal={Nature Medicine},
  year={1995},
  volume={1},
  pages={1148-1154}
}
The outcome of the first pilot study of liver-directed gene therapy is reported here. Five patients with homozygous familial hypercholesterolaemia (FH) ranging in age from 7 to 41 years were enrolled; each patient tolerated the procedure well without significant complications. Transgene expression was detected in a limited number of hepatocytes of liver tissue harvested four months after gene transfer from all five patients. Significant and prolonged reductions in low density lipoprotein (LDL… Expand
495 Citations

Paper Mentions

Interventional Clinical Trial
Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic metabolic disorder characterized by absent or severely reduced capacity to catabolize circulating LDL particles by… Expand
ConditionsHomozygous Familial Hypercholesterolemia
InterventionBiological
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