Found: 10
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Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.
- Published in:
- Journal of Clinical Investigation, 2015, v. 125, n. 2, p. 870, doi. 10.1172/JCI79213
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- Article
Exome sequencing identifies ACSF3 as a cause of combined malonic and methylmalonic aciduria.
- Published in:
- Nature Genetics, 2011, v. 43, n. 9, p. 883, doi. 10.1038/ng.908
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- Article
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice.
- Published in:
- Life Science Alliance, 2021, v. 4, n. 10, p. 1, doi. 10.26508/lsa.202101040
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- Article
Mitochondrial dysfunction in mut methylmalonic acidemia.
- Published in:
- FASEB Journal, 2009, v. 23, n. 4, p. 1252, doi. 10.1096/fj.08-121848
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- Article
Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 63, doi. 10.1002/jimd.12665
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- Article
Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1.
- Published in:
- Human Molecular Genetics, 2017, v. 26, n. 1, p. 52, doi. 10.1093/hmg/ddw367
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- Article
A Barrel of Monkeys: scAAV8 Gene Therapy for Hemophilia in Nonhuman Primates.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 826, doi. 10.1038/mt.2011.73
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- Article
Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy.
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- Molecular Therapy, 2010, v. 18, n. 1, p. 11, doi. 10.1038/mt.2009.247
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- Article
Metabolic phenotype of methylmalonic acidemia in mice andhumans: the role of skeletal muscle.
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- BMC Medical Genetics, 2007, v. 8, p. 64, doi. 10.1186/1471-2350-8-64
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- Article
Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes.
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- BMC Medical Genetics, 2007, v. 8, p. 24, doi. 10.1186/1471-2350-8-24
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- Publication type:
- Article