Congenital erythropoietic porphyria: advances in pathogenesis and treatment

Br J Haematol. 2002 Jun;117(4):779-95. doi: 10.1046/j.1365-2141.2002.03557.x.
No abstract available

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Base Sequence
  • Bone Marrow Transplantation
  • Genetic Therapy
  • Hematopoietic Stem Cell Transplantation
  • Heme / biosynthesis
  • Humans
  • Infant, Newborn
  • Mice
  • Mice, Knockout
  • Molecular Sequence Data
  • Mutation
  • Porphyria, Erythropoietic / etiology
  • Porphyria, Erythropoietic / genetics*
  • Porphyria, Erythropoietic / therapy
  • Research Design
  • Uroporphyrinogen III Synthetase / genetics

Substances

  • Heme
  • Uroporphyrinogen III Synthetase