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Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by mutations in the TYMP gene, which encodes thymidine phosphorylase (TP). TP dysfunction results in systemic thymidine (dThd) and deoxyuridine (dUrd) overload, which selectively impair mitochondr...
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| Pubblicato in: | Gene Ther |
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| Autori principali: | , , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2011
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7568345/ https://ncbi.nlm.nih.gov/pubmed/21451581 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2011.24 |
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