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

por Tanpaiboon, Pranoot, Venditti, Charles P.
Publicado 2013

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

por CHANDLER, RANDY J., VENDITTI, CHARLES P.
Publicado 2008

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

por Chandler, Randy J, Venditti, Charles P
Publicado 2009

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

por Chandler, Randy J, Venditti, Charles P
Publicado 2011

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

por Chandler, Randy J., Venditti, Charles P.
Publicado 2012

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

por Carrillo-Carrasco, Nuria, Venditti, Charles P.
Publicado 2011

Gene therapy for metabolic diseases

por Chandler, Randy J., Venditti, Charles P.
Publicado en Transl Sci Rare Dis (2016)

Methylmalonic and Propionic Acidemias: Clinical Management Update

por Fraser, Jamie L., Venditti, Charles P.
Publicado en Curr Opin Pediatr (2016)

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

por Chandler, Randy J., Venditti, Charles P.
Publicado en Hum Gene Ther (2019)

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

por Chandler, Randy J., Sands, Mark S., Venditti, Charles P.
Publicado en Hum Gene Ther (2017)

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

por Myles, Jennifer G., Manoli, Irini, Venditti, Charles P.
Publicado en Curr Opin Clin Nutr Metab Care (2018)

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

por Carrillo-Carrasco, Nuria, Chandler, Randy J., Venditti, Charles P.
Publicado en 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

por Carrillo-Carrasco, Nuria, Chandler, Randy J., Chandrasekaran, Suma, Venditti, Charles P.
Publicado 2010

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

por Schneller, Jessica L., Lee, Ciaran M., Bao, Gang, Venditti, Charles P.
Publicado en BMC Med (2017)

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

por Hauser, Natalie S, Manoli, Irini, Graf, Jennifer C, Sloan, Jennifer, Venditti, Charles P
Publicado 2011

Renal Growth in Isolated Methylmalonic Acidemia (MMA)

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

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

por Manoli, Irini, Myles, Jennifer, Sloan, Jennifer L., Shchelochkov, Oleg A., Venditti, Charles P.
Publicado en Genet Med (2015)

Response to Cunningham et al

por Manoli, Irini, Myles, Jennifer G., Sloan, Jennifer L., Shchelochkov, Oleg A., Venditti, Charles P.
Publicado en Genet Med (2016)

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

por Chandler, Randy J, LaFave, Matthew C, Varshney, Gaurav K, Burgess, Shawn M, Venditti, Charles P
Publicado en Mol Ther (2016)

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

por May, Francis J., Head, PamelaSara E., Venturoni, Leah E., Chandler, Randy J., Venditti, Charles P.
Publicado en Mol Ther Methods Clin Dev (2021)