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

by Francis J. May, PamelaSara E. Head, Leah E. Venturoni, Randy J. Chandler, Charles P. Venditti
Published 2021-06-01

Considerations of expanded carrier screening: Lessons learned from combined malonic and methylmalonic aciduria

by Marie Cosette Gabriel, Stephanie M. Rice, Jennifer L. Sloan, Matthew H. Mossayebi, Charles P. Venditti, Huda B. Al‐Kouatly
Published 2021-04-01

High‐dose hydroxocobalamin achieves biochemical correction and improvement of neuropsychiatric deficits in adults with late onset cobalamin C deficiency

by Tomoyasu Higashimoto, Alexander Y. Kim, Jessica T. Ogawa, Jennifer L. Sloan, Mohammed A. Almuqbil, Julia M. Carlson, Irini Manoli, Charles P. Venditti, Meral Gunay‐Aygun, Tao Wang
Published 2020-01-01

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

by Hye-Ri Kang, Monika Gjorgjieva, Stephanie N. Smith, Elizabeth D. Brooks, Zelin Chen, Shawn M. Burgess, Randy J. Chandler, Lauren R. Waskowicz, Kylie M. Grady, Songtao Li, Gilles Mithieux, Charles P. Venditti, Fabienne Rajas, Dwight D. Koeberl
Published 2019-12-01

Defects in muscle branched-chain amino acid oxidation contribute to impaired lipid metabolism

by Carles Lerin, Allison B. Goldfine, Tanner Boes, Manway Liu, Simon Kasif, Jonathan M. Dreyfuss, Ana Luisa De Sousa-Coelho, Grace Daher, Irini Manoli, Justin R. Sysol, Elvira Isganaitis, Niels Jessen, Laurie J. Goodyear, Kirk Beebe, Walt Gall, Charles P. Venditti, Mary-Elizabeth Patti
Published 2016-10-01

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

by Petr O. Ilyinskii, Alicia M. Michaud, Gina L. Rizzo, Christopher J. Roy, Sheldon S. Leung, Stephanie L. Elkins, Teresa Capela, Aparajita Chowdhury, Lina Li, Randy J. Chandler, Irini Manoli, Eva Andres-Mateos, Lloyd P.M. Johnston, Luk H. Vandenberghe, Charles P. Venditti, Takashi Kei Kishimoto
Published 2021-09-01

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

by Rita Ferla, Marialuisa Alliegro, Margherita Dell’Anno, Edoardo Nusco, John M. Cullen, Stephanie N. Smith, Tyra G. Wolfsberg, Patricia O’Donnell, Ping Wang, Anh-Dao Nguyen, Randy J. Chandler, Zelin Chen, Shawn M. Burgess, Charles H. Vite, Mark E. Haskins, Charles P. Venditti, Alberto Auricchio
Published 2021-03-01

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

by Ding An, Jessica L. Schneller, Andrea Frassetto, Shi Liang, Xuling Zhu, Ji-Sun Park, Matt Theisen, Sue-Jean Hong, Jenny Zhou, Raj Rajendran, Becca Levy, Rebecca Howell, Gilles Besin, Vladimir Presnyak, Staci Sabnis, Kerry E. Murphy-Benenato, E. Sathyajith Kumarasinghe, Timothy Salerno, Cosmin Mihai, Christine M. Lukacs, Randy J. Chandler, Lin T. Guey, Charles P. Venditti, Paolo G.V. Martini
Published 2017-12-01