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 em 2013

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

Por CHANDLER, RANDY J., VENDITTI, CHARLES P.
Publicado em 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 em 2009

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

Por Chandler, Randy J, Venditti, Charles P
Publicado em 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 em 2012

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

Por Carrillo-Carrasco, Nuria, Venditti, Charles P.
Publicado em 2011

Gene therapy for metabolic diseases

Por Chandler, Randy J., Venditti, Charles P.
Publicado no Transl Sci Rare Dis (2016)

Methylmalonic and Propionic Acidemias: Clinical Management Update

Por Fraser, Jamie L., Venditti, Charles P.
Publicado no Curr Opin Pediatr (2016)

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

Por Chandler, Randy J., Venditti, Charles P.
Publicado no 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 no 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 no 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 no 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 em 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 no 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 em 2011

Renal Growth in Isolated Methylmalonic Acidemia (MMA)

Por Kruszka, Paul S., Manoli, Irini, Sloan, Jennifer L., Kopp, Jeffrey B., Venditti, Charles P.
Publicado em 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 no Genet Med (2015)

Response to Cunningham et al

Por Manoli, Irini, Myles, Jennifer G., Sloan, Jennifer L., Shchelochkov, Oleg A., Venditti, Charles P.
Publicado no 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 no Mol Ther (2016)

Ion-abrasion scanning electron microscopy reveals distorted liver mitochondrial morphology in murine methylmalonic acidemia

Por Murphy, Gavin E., Lowekamp, Bradley C., Zerfas, Patricia M., Chandler, Randy J., Venditti, Charles P., Subramaniam, Sriram
Publicado em 2010