机译:Crispr-介导治疗基因组编辑对单一疾病的进展与挑战
Leon H.Charney Division of Cardiology New York University Grossman School of Medicine New York NY 10016 USA;
Leon H.Charney Division of Cardiology New York University Grossman School of Medicine New York NY 10016 USAHelen and Martin Kimmel Center for Stem Cell Biology New York University Grossman School of Medicine New York NY 10016 USADepartment of Neurology New York University Grossman School of Medicine New York NY 10016 USADepartment of Neuroscience and Physiology New York University Grossman School of Medicine New York NY 10016 USA;
gene editing; CRISPR-associated protein 9; CRISPR-Cas system; genetic disease; medical genetics; genetic therapy;