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List of works by Randy Chandler

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

scientific article

Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia ⬇️

scientific article published on February 16, 2011

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

scientific article published on January 2008

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

scientific article

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

scientific article

Gene Therapy for Metabolic Diseases

scientific article

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

scientific article published on 16 August 2019

Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery

scientific article

Genetic and genomic systems to study methylmalonic acidemia.

scientific article

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

scientific article published on February 2016

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

scientific article published on 17 October 2013

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

scientific article

Messenger RNA therapy as an option for treating metabolic disorders

scientific article published on 19 September 2019

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

scientific article

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

scientific article published on 20 January 2016

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

scientific article published on September 25, 2012

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

scientific article

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

scientific article published on 01 August 2018

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.

scientific article published in December 2017

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

scientific article published on 9 October 2003

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

scientific article

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