Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE-Reference-Cited by-同舟云学术

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Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE

Published:2011-03-31 Issue:8 Volume:18 Page:795-806
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Torres-Torronteras J,Gómez A,Eixarch H,Palenzuela L,Pizzorno G,Hirano M,Andreu A L,Barquinero J,Martí R

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/gt201124.pdf

Reference34 articles.

1. Nishino I, Spinazzola A, Hirano M . Thymidine phosphorylase gene mutations in MNGIE, a human mitochondrial disorder. Science 1999; 283: 689–692.

2. Hirano M, Nishigaki Y, Marti R . Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a disease of two genomes. Neurologist 2004; 10: 8–17.

3. Lara MC, Valentino ML, Torres-Torronteras J, Hirano M, Martí R . Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): biochemical features and therapeutic approaches. Biosci Rep 2007; 27: 151–163.

4. Halter J, Schüpbach WM, Casali C, Elhasid R, Fay K, Hammans S et al. Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach. Bone Marrow Transplant 2011; 46: 330–337.

5. Desgranges C, Razaka G, Rabaud M, Bricaud H . Catabolism of thymidine in human blood platelets: purification and properties of thymidine phosphorylase. Biochim Biophys Acta 1981; 654: 211–218.

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