Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheep.
对泰-萨克斯绵羊进行双向 AAV 基因治疗的五年疗效和安全性分析。
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI182942
Taghian Toloo, Gallagher Jillian, Bertrand Stephanie, Baker William C, Lopez Mercado Kalajan, Benatti Hector R, Hall Erin, Lopez Yvette, McElroy Abigail, McCarthy John T, Pulaparthi Sanjana, Fernau Deborah, Mather Samuel, Esteves Sophia, Diffie Elise, Gross Amanda, Lahey Hannah G, Jiang Xuntian, Parsley Elizabeth, Gately Rachael, Prestigiacomo Rachel, Johnson Siauna, Taylor Amanda, Bierfeldt Lindsey, Tuominen Susan, Koehler Jennifer, Gao Guangping, Xie Jun, Su Qin, King Robert, Gounis Matthew J, Anagnostakou Vania, Puri Ajit, Batista Ana Rita, Sena-Esteves Miguel, Martin Douglas R, Gray-Edwards Heather