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.
Chang
C C
CC
Department of Microbiology, Soochow University, Taipei, Taiwan, Republic of China.
Hsiao
K J
KJ
Chen
M L
ML
Lin
C M
CM
D018952:Antigens, CD34; D002648:Child; D015316:Genetic Therapy; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000502:physiology*; D006801:Humans; D008661:Metabolism, Inborn Errors / Q000628:therapy*; D008764:Methylmalonic Acid / Q000097:blood* / Q000378:metabolism; D008765:Methylmalonyl-CoA Mutase / Q000235:genetics*; D010641:Phenotype; D012190:Retroviridae / Q000235:genetics; D020133:Reverse Transcriptase Polymerase Chain Reaction
D018952:Antigens, CD34; D008764:Methylmalonic Acid; D008765:Methylmalonyl-CoA Mutase