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Correction of Methylmalonic Aciduria In Vivo Using a Codon-Optimized Lentiviral Vector
Methylmalonic aciduria is a rare disorder of organic acid metabolism with limited therapeutic options, resulting in high morbidity and mortality. Positive results from combined liver/kidney transplantation suggest, however, that metabolic sink therapy may be efficacious. Gene therapy offers a more a...
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| Главные авторы: | , , , , , |
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| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
Mary Ann Liebert, Inc.
2014
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4064729/ https://ncbi.nlm.nih.gov/pubmed/24568291 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.111 |
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