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

par Tanpaiboon, Pranoot, Venditti, Charles P.
Publié 2013

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

par CHANDLER, RANDY J., VENDITTI, CHARLES P.
Publié 2008

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

par Chandler, Randy J, Venditti, Charles P
Publié 2009

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

par Chandler, Randy J, Venditti, Charles P
Publié 2011

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

par Chandler, Randy J., Venditti, Charles P.
Publié 2012

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

par Carrillo-Carrasco, Nuria, Venditti, Charles P.
Publié 2011

Gene therapy for metabolic diseases

par Chandler, Randy J., Venditti, Charles P.
Publié dans Transl Sci Rare Dis (2016)

Methylmalonic and Propionic Acidemias: Clinical Management Update

par Fraser, Jamie L., Venditti, Charles P.
Publié dans Curr Opin Pediatr (2016)

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

par Chandler, Randy J., Venditti, Charles P.
Publié dans Hum Gene Ther (2019)

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

par Chandler, Randy J., Sands, Mark S., Venditti, Charles P.
Publié dans Hum Gene Ther (2017)

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

par Myles, Jennifer G., Manoli, Irini, Venditti, Charles P.
Publié dans Curr Opin Clin Nutr Metab Care (2018)

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

par Carrillo-Carrasco, Nuria, Chandler, Randy J., Venditti, Charles P.
Publié dans 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

par Carrillo-Carrasco, Nuria, Chandler, Randy J., Chandrasekaran, Suma, Venditti, Charles P.
Publié 2010

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

par Schneller, Jessica L., Lee, Ciaran M., Bao, Gang, Venditti, Charles P.
Publié dans BMC Med (2017)

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

par Hauser, Natalie S, Manoli, Irini, Graf, Jennifer C, Sloan, Jennifer, Venditti, Charles P
Publié 2011

Renal Growth in Isolated Methylmalonic Acidemia (MMA)

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

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

par Manoli, Irini, Myles, Jennifer, Sloan, Jennifer L., Shchelochkov, Oleg A., Venditti, Charles P.
Publié dans Genet Med (2015)

Response to Cunningham et al

par Manoli, Irini, Myles, Jennifer G., Sloan, Jennifer L., Shchelochkov, Oleg A., Venditti, Charles P.
Publié dans Genet Med (2016)

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

par Chandler, Randy J, LaFave, Matthew C, Varshney, Gaurav K, Burgess, Shawn M, Venditti, Charles P
Publié dans Mol Ther (2016)

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

par May, Francis J., Head, PamelaSara E., Venturoni, Leah E., Chandler, Randy J., Venditti, Charles P.
Publié dans Mol Ther Methods Clin Dev (2021)