Towards metabolic sink therapy for mut methylmalonic acidaemia: retrovirus-mediated transfer of the human methylmalonyl-CoA mutase cDNA into peripheral blood progenitor cells of a child with mut methylmalonic acidaemia

J Inherit Metab Dis. 1999 Dec;22(8):951-2. doi: 10.1023/a:1005624415344.
No abstract available

Publication types

  • Case Reports

MeSH terms

  • Antigens, CD34
  • Child
  • Genetic Therapy*
  • Hematopoietic Stem Cell Transplantation*
  • Hematopoietic Stem Cells / physiology*
  • Humans
  • Metabolism, Inborn Errors / therapy*
  • Methylmalonic Acid / blood*
  • Methylmalonic Acid / metabolism
  • Methylmalonyl-CoA Mutase / genetics*
  • Phenotype
  • Retroviridae / genetics
  • Reverse Transcriptase Polymerase Chain Reaction

Substances

  • Antigens, CD34
  • Methylmalonic Acid
  • Methylmalonyl-CoA Mutase