Messenger RNA therapy as an option for treating metabolic disorders

Tekijä Chandler, Randy J.
Julkaisussa Proc Natl Acad Sci U S A (2019)

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

Tekijä CHANDLER, RANDY J., VENDITTI, CHARLES P.
Julkaistu 2008

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

Tekijä Chandler, Randy J, Venditti, Charles P
Julkaistu 2009

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

Tekijä Chandler, Randy J, Venditti, Charles P
Julkaistu 2011

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

Tekijä Chandler, Randy J., Venditti, Charles P.
Julkaistu 2012

Gene therapy for metabolic diseases

Tekijä Chandler, Randy J., Venditti, Charles P.
Julkaisussa Transl Sci Rare Dis (2016)

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

Tekijä Chandler, Randy J., Venditti, Charles P.
Julkaisussa Hum Gene Ther (2019)

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

Tekijä Chandler, Randy J., Sands, Mark S., Venditti, Charles P.
Julkaisussa Hum Gene Ther (2017)

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

Tekijä Carrillo-Carrasco, Nuria, Chandler, Randy J., Venditti, Charles P.
Julkaisussa 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

Tekijä Carrillo-Carrasco, Nuria, Chandler, Randy J., Chandrasekaran, Suma, Venditti, Charles P.
Julkaistu 2010

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

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

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

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

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

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

Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes

Tekijä Chandler, Randy J, Tsai, Matthew S, Dorko, Kenneth, Sloan, Jennifer, Korson, Mark, Freeman, Richard, Strom, Stephen, Venditti, Charles P
Julkaistu 2007

Mitochondrial dysfunction in mut methylmalonic acidemia

Tekijä Chandler, Randy J., Zerfas, Patricia M., Shanske, Sara, Sloan, Jennifer, Hoffmann, Victoria, DiMauro, Salvatore, Venditti, Charles P.
Julkaistu 2009

Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia

Tekijä Chandler, Randy J., Chandrasekaran, Suma, Carrillo-Carrasco, Nuria, Senac, Julien S., Hofherr, Sean E., Barry, Michael A., Venditti, Charles P.
Julkaistu 2011

Liver-Directed Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Lethal Murine Model of Citrullinemia Type 1

Tekijä Chandler, Randy J., Tarasenko, Tatiana N., Cusmano-Ozog, Kristina, Sun, Qin, Sutton, V. Reid, Venditti, Charles P., McGuire, Peter J.
Julkaistu 2013

Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle

Tekijä Chandler, Randy J, Sloan, Jennifer, Fu, Hong, Tsai, Matthew, Stabler, Sally, Allen, Robert, Kaestner, Klaus H, Kazazian, Haig H, Venditti, Charles P
Julkaistu 2007

Propionyl-CoA and Adenosylcobalamin Metabolism in C. elegans : Evidence for a Role of Methylmalonyl-CoA Epimerase in Intermediary Metabolism

Tekijä Chandler, Randy J., Aswani, Vijay, Tsai, Matthew S., Falk, Marni, Wehrli, Natasha, Stabler, Sally, Allen, Robert, Sedensky, Margaret, Kazazian, Haig H., Venditti, Charles P.
Julkaistu 2006

Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia

Tekijä Harrington, Elizabeth A., Sloan, Jennifer L., Manoli, Irini, Chandler, Randy J., Schneider, Mark, McGuire, Peter J., Calcedo, Roberto, Wilson, James M., Venditti, Charles P.
Julkaisussa Hum Gene Ther (2016)