ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models

Autor: Wechsler, Thomas, DeKelver, Russell, Laoharawee, Kanut, Ou, Li, Tom, Susan, Radecke, Robert, Sproul, Scott, Koniar, Brenda L., Podetz-Perdersen, Kelly, Cooksley, Renee D., Meyer, Kathleen, McIvor, Scott R., Chester, Whitley B., Holmes, Michael C.
Zdroj: In Molecular Genetics and Metabolism February 2018 123(2):S146-S147
Databáze: ScienceDirect