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Molecular Therapy : the Journal of the American Society of Gene Therapy|October 29, 2009
Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapyRandy J Chandler, Charles P VendittiHuman Gene Therapy|December 7, 2007
Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemiaRandy J Chandler, Charles P VendittiJournal of Inherited Metabolic Disease|August 2, 2023
Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemiaRandy J Chandler, Charles P VendittiMolecular Genetics and Metabolism|October 11, 2012
Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)Randy J Chandler, Charles P VendittiHuman Gene Therapy|July 16, 2019
Gene Therapy for Methylmalonic Acidemia: Past, Present, and FutureRandy J Chandler, Charles P VendittiTranslational Science of Rare Diseases|November 18, 2016
Gene Therapy for Metabolic DiseasesRandy J Chandler, Charles P VendittiHuman Gene Therapy|March 16, 2017
Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal ModelsRandy J Chandler, Mark S Sands, Charles P VendittiJournal of Inherited Metabolic Disease|July 13, 2011
Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and managementNuria Carrillo-Carrasco, Randy J Chandler, Charles P VendittiHuman Gene Therapy|May 22, 2010
Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correctionNuria Carrillo-Carrasco, Randy J Chandler, Suma Chandrasekaran, et al.Molecular Therapy. Methods & Clinical Development|June 25, 2021
Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemiaFrancis J May, PamelaSara E Head, Leah E Venturoni, et al.Pageof 9