Randy J. Chandler

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