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Gene Therapy for Metabolic DiseasesLiver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correctionMetabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscleCombined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management.Genetic and genomic systems to study methylmalonic acidemia.A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.Exome sequencing identifies ACSF3 as a cause of combined malonic and methylmalonic aciduria.Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene deliveryGenotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia.Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1.Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models.Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.Messenger RNA therapy as an option for treating metabolic disordersSystemic Messenger RNA Therapy as a Treatment for Methylmalonic AcidemiaGene Therapy for Methylmalonic Acidemia: Past, Present, and Future
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description
Forscher
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investigador
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researcher
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ricercatore
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wetenschapper
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研究員
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研究者
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name
Randy Chandler
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Randy Chandler
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Randy Chandler
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Randy Chandler
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Randy Chandler
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Randy Chandler
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P496
0000-0002-6047-5230