Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies

por Moffit, Jeffrey S., Blanset, Diann L., Lynch, Jessica L., MacLachlan, Timothy K., Meyer, Kathleen E., Ponce, Rafael, Whiteley, Laurence O.
Publicado 2022

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

por Laoharawee, Kanut, DeKelver, Russell C., Podetz-Pedersen, Kelly M., Rohde, Michelle, Sproul, Scott, Nguyen, Hoang-Oanh, Nguyen, Tam, St. Martin, Susan J., Ou, Li, Tom, Susan, Radeke, Robert, Meyer, Kathleen E., Holmes, Michael C., Whitley, Chester B., Wechsler, Thomas, McIvor, R. Scott
Publicado 2018

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

por Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michael J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Meyer, Kathleen E., Holmes, Michael C., McIvor, R. Scott, Wechsler, Thomas, Whitley, Chester B.
Publicado 2019