Randy J. Chandler

Propionyl-CoA and adenosylcobalamin metabolism in Caenorhabditis elegans: evidence for a role of methylmalonyl-CoA epimerase in intermediary metabolism.

Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes.

Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle.

Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia.

Mitochondrial dysfunction in mut methylmalonic acidemia.

Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy.

Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction.

Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia.

A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.

Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management.

Exome sequencing identifies ACSF3 as a cause of combined malonic and methylmalonic aciduria.

Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA).

Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia.

Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1.

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.

Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia.

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1.

Gene Therapy for Metabolic Diseases.

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models.

FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.

Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future.

Messenger RNA therapy as an option for treating metabolic disorders.

Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.

The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup.

Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia.

Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice.

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia.

Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing.

Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9.

From Puppies to adults: editing of hepatocytes in a canine model of glycogen storage disease type Ia.

Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia.

Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia.