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Short-Term Rescue of Neonatal Lethality in a Mouse Model of Propionic Acidemia by Gene Therapy
Propionic acidemia (PA) is a metabolic disorder that causes mental retardation and that can be fatal if untreated. PA is inherited in an autosomal recessive fashion involving mutations in PCCA or PCCB encoding the α and β subunits of propionyl-CoA carboxylase (PCC). Current treatment is based on die...
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| Main Authors: | , , , , , |
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| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
Mary Ann Liebert, Inc.
2009
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2922073/ https://ncbi.nlm.nih.gov/pubmed/19025475 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2008.158 |
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