Noncompaction of the ventricular myocardium and hydrops fetalis in Cobalamin C disease: Response to letter “Disappearance of congenital noncompaction in hereditary cobalamin-C-defi...

per Tanpaiboon, Pranoot, Venditti, Charles P.
Publicat 2013

Adenovirus-Mediated Gene Delivery Rescues a Neonatal Lethal Murine Model of mut(0) Methylmalonic Acidemia

per CHANDLER, RANDY J., VENDITTI, CHARLES P.
Publicat 2008

Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy

per Chandler, Randy J, Venditti, Charles P
Publicat 2009

A Barrel of Monkeys: scAAV8 Gene Therapy for Hemophilia in Nonhuman Primates

per Chandler, Randy J, Venditti, Charles P
Publicat 2011

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

per Chandler, Randy J., Venditti, Charles P.
Publicat 2012

Combined methylmalonic acidemia and homocystinuria, cblC type. II. Complications, pathophysiology, and outcomes

per Carrillo-Carrasco, Nuria, Venditti, Charles P.
Publicat 2011

Gene therapy for metabolic diseases

per Chandler, Randy J., Venditti, Charles P.
Publicat a Transl Sci Rare Dis (2016)

Methylmalonic and Propionic Acidemias: Clinical Management Update

per Fraser, Jamie L., Venditti, Charles P.
Publicat a Curr Opin Pediatr (2016)

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

per Chandler, Randy J., Venditti, Charles P.
Publicat a Hum Gene Ther (2019)

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

per Chandler, Randy J., Sands, Mark S., Venditti, Charles P.
Publicat a Hum Gene Ther (2017)

Effects of Medical Food Leucine Content in the Management of Methylmalonic and Propionic Acidemias

per Myles, Jennifer G., Manoli, Irini, Venditti, Charles P.
Publicat a Curr Opin Clin Nutr Metab Care (2018)

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

per Carrillo-Carrasco, Nuria, Chandler, Randy J., Venditti, Charles P.
Publicat a J Inherit Metab Dis (2011)

Liver-Directed Recombinant Adeno-Associated Viral Gene Delivery Rescues a Lethal Mouse Model of Methylmalonic Acidemia and Provides Long-Term Phenotypic Correction

per Carrillo-Carrasco, Nuria, Chandler, Randy J., Chandrasekaran, Suma, Venditti, Charles P.
Publicat 2010

Genome editing for inborn errors of metabolism: advancing towards the clinic

per Schneller, Jessica L., Lee, Ciaran M., Bao, Gang, Venditti, Charles P.
Publicat a BMC Med (2017)

Variable dietary management of methylmalonic acidemia: metabolic and energetic correlations(1)(2)(3)(4)

per Hauser, Natalie S, Manoli, Irini, Graf, Jennifer C, Sloan, Jennifer, Venditti, Charles P
Publicat 2011

Renal Growth in Isolated Methylmalonic Acidemia (MMA)

per Kruszka, Paul S., Manoli, Irini, Sloan, Jennifer L., Kopp, Jeffrey B., Venditti, Charles P.
Publicat 2013

A critical reappraisal of dietary practices in methylmalonic acidemia raises concerns about the safety of medical foods. Part 1: Isolated methylmalonic acidemias (MMA)

per Manoli, Irini, Myles, Jennifer, Sloan, Jennifer L., Shchelochkov, Oleg A., Venditti, Charles P.
Publicat a Genet Med (2015)

Response to Cunningham et al

per Manoli, Irini, Myles, Jennifer G., Sloan, Jennifer L., Shchelochkov, Oleg A., Venditti, Charles P.
Publicat a Genet Med (2016)

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

per Chandler, Randy J, LaFave, Matthew C, Varshney, Gaurav K, Burgess, Shawn M, Venditti, Charles P
Publicat a Mol Ther (2016)

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

per May, Francis J., Head, PamelaSara E., Venturoni, Leah E., Chandler, Randy J., Venditti, Charles P.
Publicat a Mol Ther Methods Clin Dev (2021)