Literature DB >> 10604156

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.

C C Chang1, K J Hsiao, M L Chen, C M Lin.   

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Year:  1999        PMID： 10604156     DOI： 10.1023/a:1005624415344

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN： 0141-8955            Impact factor:   4.982


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  3 in total

1.  Towards metabolic sink therapy for mut methylmalonic acidaemia: correction of methylmalonyl-CoA mutase deficiency in T lymphocytes from a mut methylmalonic acidaemia child by retroviral-mediated gene transfer.

Authors:  C C Chang; K J Hsiao; Y M Lee; C M Lin
Journal:  J Inherit Metab Dis       Date:  1999-10       Impact factor: 4.982

2.  Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation.

Authors:  C E Dunbar; D B Kohn; R Schiffmann; N W Barton; J A Nolta; J A Esplin; M Pensiero; Z Long; C Lockey; R V Emmons; S Csik; S Leitman; C B Krebs; C Carter; R O Brady; S Karlsson
Journal:  Hum Gene Ther       Date:  1998-11-20       Impact factor: 5.695

3.  T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

Authors:  D B Kohn; M S Hershfield; D Carbonaro; A Shigeoka; J Brooks; E M Smogorzewska; L W Barsky; R Chan; F Burotto; G Annett; J A Nolta; G Crooks; N Kapoor; M Elder; D Wara; T Bowen; E Madsen; F F Snyder; J Bastian; L Muul; R M Blaese; K Weinberg; R Parkman
Journal:  Nat Med       Date:  1998-07       Impact factor: 53.440
  3 in total

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