Scott Sproul

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Rajiv P. Sharma, Xavier M. Anguela, Yannick Doyon et al. · Blood · 2015 · 298 citations · Full text

Life Sciences, Genetics, Molecular Biology +16

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 et al. · Molecular Therapy · 2018 · 125 citations · Full text

Life Sciences, Genetics, Molecular Genetics +15

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Li Ou, Russell C. DeKelver, Michelle Rohde et al. · Molecular Therapy · 2018 · 85 citations · Full text

Life Sciences, Genetics, Molecular Genetics +14

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, Silvère Pagant et al. · Molecular Therapy — Methods & Clinical Development · 2020 · 55 citations · Full text

Life Sciences, Genetics, Molecular Genetics +16

ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice

Silvère Pagant, Marshall W. Huston, Luciana Moreira et al. · Molecular Therapy · 2021 · 23 citations · Full text

Life Sciences, Molecular Biology, Molecular Genetics +16