Publications by authors named "Scott Sproul"

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ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice.
Silvere Pagant, Marshall W Huston, Luciana Moreira, Lin Gan, Susan St Martin, Scott Sproul

Mol Ther· November 2021

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AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
Makiko Yasuda, Marshall W Huston, Silvere Pagant, Lin Gan, Susan St Martin, Scott Sproul

Mol Ther Methods Clin Dev· September 2020

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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
Li Ou, Russell C DeKelver, Michelle Rohde, Susan Tom, Robert Radeke, Scott Sproul

Mol Ther· January 2019

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Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
Kanut Laoharawee, Russell C DeKelver, Kelly M Podetz-Pedersen, Michelle Rohde, Scott Sproul

Mol Ther· April 2018

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In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
Rajiv Sharma, Xavier M Anguela, Yannick Doyon, Thomas Wechsler, Russell C DeKelver, Scott Sproul

Blood· October 2015

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