Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling by Randy J. Chandler

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From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia by Randy J. Chandler

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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

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Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9 by Randy J. Chandler, Giovanni Di Pasquale, Jennifer L. Sloan, Samantha McCoy, Brandon T. Hubbard, Tina M. Kilts, Irini Manoli, John A. Chiorini, Charles P. Venditti

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Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia by Randy J. Chandler, Giovanni Di Pasquale, Eun-Young Choi, David Chang, Stephanie N. Smith, Jennifer L. Sloan, Victoria Hoffmann, Lina Li, John A. Chiorini, Charles P. Venditti

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In vivo genome editing at the albumin locus to treat methylmalonic acidemia by Jessica L. Schneller, Ciaran M. Lee, Leah E. Venturoni, Randy J. Chandler, Ang Li, Sangho Myung, Thomas J. Cradick, Ayrea E. Hurley, William R. Lagor, Gang Bao, Charles P. Venditti

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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

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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

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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

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