Stem Cell Related Patent Number US6312957

Genetic modification of primate hemopoietic repopulating stem cells

Einerhand, Markus Peter Wilhelmus; Amsterdam, Netherlands

This invention describes the transfection of primate pluripotent hematopoietic stem cells with an adeno-associated viral vector, methods and uses thereof. Described herein is the genetic modification of pluripotent hemopoietic stem cells of primates (P-PHSC) by transduction of the P-PHSCs with a recombinant adeno-associated virus (AAV). Claims of the invention include a tile genome of the recombinant AAV comprised of a DNA sequence flanked by the inverted terminal repeats (ITR) of AAV, such that the DNA sequence consists of regulatory sequences which are functional in hemopoietic cells and a sequence coding for a protein or RNA with a therapeutic property when introduced into hemopoietic cells. Further disclosed are the means by which such DNA sequences are the human lysosomal glococerebrosidase gene, a globin gene from the human beta-globin gene cluster, a DNA sequence encoding an RNA or protein with anti-viral activity, the alpha1-antitrypsin gene and the human multidrug resistance gene I (MDRI). Human therapeutic applications include the treatment of diseases such as sickle cell anaemia.

Application Number: US1999000326032

Date Filed: 06/04/99

Dated Published: 11/06/01

Assignee: Introgene B.V. Netherlands