Randy J. Chandler

Metabolic Medicine Branch, National Institutes of Health

Affiliated withNational Institutes of Health

Research Area

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JoVE Journal Publications

ArticleTotal : 1	Year
Publication title	2024

Other Publications

Article	Year
Propionyl-CoA and adenosylcobalamin metabolism in Caenorhabditis elegans: evidence for a role of methylmalonyl-CoA epimerase in intermediary metabolism. Molecular genetics and metabolism\| PubMed ID: 16843692	2006
Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes. BMC medical genetics\| PubMed ID: 17470278	2007
Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle. BMC medical genetics\| PubMed ID: 17937813	2007
Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia. Human gene therapy\| PubMed ID: 18052792	2008
Mitochondrial dysfunction in mut methylmalonic acidemia. FASEB journal : official publication of the Federation of American Societies for Experimental Biology\| PubMed ID: 19088183	2009
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\| PubMed ID: 19861951	2010
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\| PubMed ID: 20486773	2010
Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia. Human gene therapy\| PubMed ID: 20950151	2011
A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 21532610	2011
Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management. Journal of inherited metabolic disease\| PubMed ID: 21748409	2012
Exome sequencing identifies ACSF3 as a cause of combined malonic and methylmalonic aciduria. Nature genetics\| PubMed ID: 21841779	2011
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\| PubMed ID: 23046887	2012
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\| PubMed ID: 23898205	2013
Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1. Gene therapy\| PubMed ID: 24131980	2013
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. The Journal of clinical investigation\| PubMed ID: 25607839	2015
Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia. Human gene therapy\| PubMed ID: 26790480	2016
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\| PubMed ID: 26906613	2016
Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1. Human molecular genetics\| PubMed ID: 27798114	2017
Gene Therapy for Metabolic Diseases. Translational science of rare diseases\| PubMed ID: 27853673	2016
Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models. Human gene therapy\| PubMed ID: 28293963	2017
FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia. JCI insight\| PubMed ID: 30518688	2018
Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future. Human gene therapy\| PubMed ID: 31303064	2019
Messenger RNA therapy as an option for treating metabolic disorders. Proceedings of the National Academy of Sciences of the United States of America\| PubMed ID: 31537746	2019
Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia. Hepatology (Baltimore, Md.)\| PubMed ID: 32976669	2021
The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup. Human gene therapy\| PubMed ID: 32993373	2020
Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia. Molecular therapy. Methods & clinical development\| PubMed ID: 34169115	2021
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice. Life science alliance\| PubMed ID: 34407999	2021
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia. Molecular therapy. Methods & clinical development\| PubMed ID: 34485611	2021
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\| PubMed ID: 35868241	2022
Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9. Molecular therapy. Methods & clinical development\| PubMed ID: 36186952	2022
From Puppies to adults: editing of hepatocytes in a canine model of glycogen storage disease type Ia. Molecular therapy. Methods & clinical development\| PubMed ID: 37206367	2023
Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia. Journal of inherited metabolic disease\| PubMed ID: 37530705	2024
Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia. Molecular therapy. Methods & clinical development\| PubMed ID: 37746248	2023