Randy J. Chandler Metabolic Medicine Branch, National Human Genome Institute National Institutes of Health Biography Publications Institution JoVE Articles Randy J. Chandler has not added a biography. If you are Randy J. Chandler and would like to personalize this page please email our Author Liaison for assistance. Publications Gene Therapy for Organic Acidemias: Lessons Learned from Methylmalonic and Propionic Acidemia Journal of Inherited Metabolic Disease. Jan, 2024 | Pubmed ID: 37530705 Systemic Gene Therapy Using an AAV44.9 Vector Rescues a Neonatal Lethal Mouse Model of Propionic Acidemia Molecular Therapy. Methods & Clinical Development. Sep, 2023 | Pubmed ID: 37746248 From Puppies to Adults: Editing of Hepatocytes in a Canine Model of Glycogen Storage Disease Type Ia Molecular Therapy. Methods & Clinical Development. Jun, 2023 | Pubmed ID: 37206367 Systemic Gene Therapy for Methylmalonic Acidemia Using the Novel Adeno-associated Viral Vector 44.9 Molecular Therapy. Methods & Clinical Development. Dec, 2022 | Pubmed ID: 36186952 Growth Advantage of Corrected Hepatocytes in a Juvenile Model of Methylmalonic Acidemia Following Liver Directed Adeno-associated Viral Mediated Nuclease-free Genome Editing Molecular Genetics and Metabolism. 2022 | Pubmed ID: 35868241 ImmTOR Nanoparticles Enhance AAV Transgene Expression After Initial and Repeat Dosing in a Mouse Model of Methylmalonic Acidemia Molecular Therapy. Methods & Clinical Development. Sep, 2021 | Pubmed ID: 34485611 Improved Systemic AAV Gene Therapy with a Neurotrophic Capsid in Niemann-Pick Disease Type C1 Mice Life Science Alliance. Oct, 2021 | Pubmed ID: 34407999 Central Nervous System-targeted Adeno-associated Virus Gene Therapy in Methylmalonic Acidemia Molecular Therapy. Methods & Clinical Development. Jun, 2021 | Pubmed ID: 34169115 Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia Hepatology (Baltimore, Md.). Jun, 2021 | Pubmed ID: 32976669 The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup Human Gene Therapy. Oct, 2020 | Pubmed ID: 32993373 Messenger RNA Therapy As an Option for Treating Metabolic Disorders Proceedings of the National Academy of Sciences of the United States of America. Oct, 2019 | Pubmed ID: 31537746 Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future Human Gene Therapy. Oct, 2019 | Pubmed ID: 31303064 FGF21 Underlies a Hormetic Response to Metabolic Stress in Methylmalonic Acidemia JCI Insight. Dec, 2018 | Pubmed ID: 30518688 Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models Human Gene Therapy. Apr, 2017 | Pubmed ID: 28293963 Systemic AAV9 Gene Therapy Improves the Lifespan of Mice with Niemann-Pick Disease, Type C1 Human Molecular Genetics. Jan, 2017 | Pubmed ID: 27798114 Gene Therapy for Metabolic Diseases Translational Science of Rare Diseases. 2016 | Pubmed ID: 27853673 Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy? Molecular Therapy : the Journal of the American Society of Gene Therapy. Feb, 2016 | Pubmed ID: 26906613 Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with Mut Methylmalonic Acidemia Human Gene Therapy. May, 2016 | Pubmed ID: 26790480 Vector Design Influences Hepatic Genotoxicity After Adeno-associated Virus Gene Therapy The Journal of Clinical Investigation. Feb, 2015 | Pubmed ID: 25607839 Liver-directed Adeno-associated Virus Serotype 8 Gene Transfer Rescues a Lethal Murine Model of Citrullinemia Type 1 Gene Therapy. Dec, 2013 | Pubmed ID: 24131980 Targeting Proximal Tubule Mitochondrial Dysfunction Attenuates the Renal Disease of Methylmalonic Acidemia Proceedings of the National Academy of Sciences of the United States of America. Aug, 2013 | Pubmed ID: 23898205 Pre-clinical Efficacy and Dosing of an AAV8 Vector Expressing Human Methylmalonyl-CoA Mutase in a Murine Model of Methylmalonic Acidemia (MMA) Molecular Genetics and Metabolism. Nov, 2012 | Pubmed ID: 23046887 Combined Methylmalonic Acidemia and Homocystinuria, CblC Type. I. Clinical Presentations, Diagnosis and Management Journal of Inherited Metabolic Disease. Jan, 2012 | Pubmed ID: 21748409 Exome Sequencing Identifies ACSF3 As a Cause of Combined Malonic and Methylmalonic Aciduria Nature Genetics. Aug, 2011 | Pubmed ID: 21841779 A Barrel of Monkeys: ScAAV8 Gene Therapy for Hemophilia in Nonhuman Primates Molecular Therapy : the Journal of the American Society of Gene Therapy. May, 2011 | Pubmed ID: 21532610 Adeno-associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia Human Gene Therapy. Apr, 2011 | Pubmed ID: 20950151 Liver-directed Recombinant Adeno-associated Viral Gene Delivery Rescues a Lethal Mouse Model of Methylmalonic Acidemia and Provides Long-term Phenotypic Correction Human Gene Therapy. Sep, 2010 | Pubmed ID: 20486773 Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno-associated Viral Gene Therapy Molecular Therapy : the Journal of the American Society of Gene Therapy. Jan, 2010 | Pubmed ID: 19861951 Mitochondrial Dysfunction in Mut Methylmalonic Acidemia FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology. Apr, 2009 | Pubmed ID: 19088183 Adenovirus-mediated Gene Delivery Rescues a Neonatal Lethal Murine Model of Mut(0) Methylmalonic Acidemia Human Gene Therapy. Jan, 2008 | Pubmed ID: 18052792 Metabolic Phenotype of Methylmalonic Acidemia in Mice and Humans: the Role of Skeletal Muscle BMC Medical Genetics. Oct, 2007 | Pubmed ID: 17937813 Adenoviral-mediated Correction of Methylmalonyl-CoA Mutase Deficiency in Murine Fibroblasts and Human Hepatocytes BMC Medical Genetics. Apr, 2007 | Pubmed ID: 17470278 Propionyl-CoA and Adenosylcobalamin Metabolism in Caenorhabditis Elegans: Evidence for a Role of Methylmalonyl-CoA Epimerase in Intermediary Metabolism Molecular Genetics and Metabolism. 2006 | Pubmed ID: 16843692 Systemic Treatment for Postnatal, Juvenile, and Runted Adult Mice by Retrobulbar Sinus Injection Darwin Romero1, Randy J. Chandler1 1Metabolic Medicine Branch, National Human Genome Institute, National Institutes of Health JoVE 66809 Genetics
Systemic Treatment for Postnatal, Juvenile, and Runted Adult Mice by Retrobulbar Sinus Injection Darwin Romero1, Randy J. Chandler1 1Metabolic Medicine Branch, National Human Genome Institute, National Institutes of Health JoVE 66809 Genetics