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

被引：7

作者：

Chang, CC

Hsiao, KJ

Chen, ML

Lin, CM ^{[1
]}

机构：

[1] Soochow Univ, Dept Microbiol, Taipei 11102, Taiwan

[2] Natl Yang Ming Univ, Inst Genet, Taipei 112, Taiwan

[3] Vet Gen Hosp, Dept Med Res, Taipei 11217, Taiwan

来源：

JOURNAL OF INHERITED METABOLIC DISEASE | 1999年 / 22卷 / 08期

关键词：

D O I：

10.1023/A:1005624415344

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

[No abstract available]

引用

页码：951 / 952

页数：2

共 3 条

[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 [J].

Chang, CC ;

Hsiao, KJ ;

Lee, YM ;

Lin, CM .

JOURNAL OF INHERITED METABOLIC DISEASE, 1999, 22 (07) :773-787

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

Dunbar, CE ;

Kohn, DB ;

Schiffmann, R ;

Barton, NW ;

Nolta, JA ;

Esplin, JA ;

Pensiero, M ;

Long, ZF ;

Lockey, C ;

Emmons, RVB ;

Csik, S ;

Leitman, S ;

Krebs, CB ;

Carter, C ;

Brady, RO ;

Karlsson, S .

HUMAN GENE THERAPY, 1998, 9 (17) :2629-2640

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

Kohn, DB ;

Hershfield, MS ;

Carbonaro, D ;

Shigeoka, A ;

Brooks, J ;

Smogorzewska, EM ;

Barsky, LW ;

Chan, R ;

Burotto, F ;

Annett, G ;

Nolta, JA ;

Crooks, G ;

Kapoor, N ;

Elder, M ;

Wara, D ;

Bowen, T ;

Madsen, E ;

Snyder, FF ;

Bastian, J ;

Muul, L ;

Blaese, RM ;

Weinberg, K ;

Parkman, R .

NATURE MEDICINE, 1998, 4 (07) :775-780

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