GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES
摘要
The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
申请公布号
CA2960209(A1)
申请公布日期
2016.03.10
申请号
CA20152960209
申请日期
2015.09.04
申请人
MEMORIAL SLOAN-KETTERING CANCER CENTER;UNIVERSITY OF WASHINGTON