Gene therapy enters the pharma market: the short story of a long journey

EMBO Mol Med. 2013 Jan;5(1):1-3. doi: 10.1002/emmm.201202291.
No abstract available

MeSH terms

  • Dependovirus / genetics
  • Drug Approval
  • European Union
  • Gene Expression
  • Genetic Therapy / trends*
  • Genetic Vectors
  • Humans
  • Hyperlipoproteinemia Type I / enzymology
  • Hyperlipoproteinemia Type I / genetics
  • Hyperlipoproteinemia Type I / therapy*
  • Lipoprotein Lipase / deficiency
  • Lipoprotein Lipase / genetics
  • Mutation
  • Rare Diseases / enzymology
  • Rare Diseases / genetics
  • Rare Diseases / therapy
  • Recombinant Proteins / genetics
  • Triglycerides / blood

Substances

  • Recombinant Proteins
  • Triglycerides
  • LPL protein, human
  • Lipoprotein Lipase