公开(公告)号：WO2016183593A2

公开(公告)日：2016-11-17

申请号：PCT/US2016/032785

申请日：2016-05-16

申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

发明人： WANG, Aijun ,  MIGUELINO, Maricel ,  POWELL, Jerry

IPC分类号： A61K48/00 ,  C12N15/86

CPC分类号： A61K48/0075 ,  A61K48/0083 ,  C12N15/86 ,  C12N2740/15043

摘要： Compositions and methods are provided to treat or prevent a genetic disease or disorder, e.g., Hemophilia A, in a subject by administering to the subject an effective amount of a polynucleotide encoding a nucleic acid encoding a functional Factor VIII polypeptide in a pre-term placenta-derived stem cell. The subject to be treated can be a fetus and the cell is a pre-term chorionic villus tissue derived stem cell (CSC) that has been isolated from the placenta before the baby is born.

摘要翻译： 提供组合物和方法以通过向受试者施用有效量的编码在术前胎盘中编码功能性因子VIII多肽的核酸的多核苷酸来治疗或预防受试者中的遗传疾病或病症，例如血友病A 衍生的干细胞。 待治疗的对象可以是胎儿，并且细胞是在婴儿出生之前已经从胎盘分离出的术前绒毛膜绒毛组织来源的干细胞（CSC）。