Patent ID: 9226942
Filing Date: 2016-01-05
CPC Classification: A61K,A61P,C12N,C12Q,G01N

Claim Text:
1. A method of treating primary or secondary bone marrow failure syndrome, the method comprising: obtaining hematopoietic stem cells from a subject that has primary or secondary bone marrow failure syndrome; detecting a higher expression level of a gene encoding VentX in the hematopoietic stem cells as compared to that in control hematopoietic stem cells; generating expanded hematopoietic stem cells by (a) contacting isolated hematopoietic stem cells with an IRNA agent having the sequence of SEQ ID NO: 5 that inhibits the expression or activation of a VentX polypeptide, and (b) incubating the isolated hematopoietic stem cells thus contacted under conditions allowing expansion of the isolated cells, whereby expanded hematopoietic stem cells are generated; and administering to the subject an effective amount of the expanded hematopoietic stem cells.