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Stem Cell Related Patent Number US6312957
| Title: | Genetic modification of primate hemopoietic repopulating stem cells | | Inventors: | Einerhand, Markus Peter Wilhelmus; Amsterdam, Netherlands | | Summary: | 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. | | Abstract: | Genetic modification of pluripotent hemopoietic stem cells of primates (P-PHSC) by transduction of P-PHSC with a recombinant adeno-associated virus (AAV). Tile genomc of the recombinant AAV comprises a DNA sequence flanked by the inverted terminal repeats (ITR) of AAV. The DNA sequence will normally comprise regulatory sequences which are functional in hemopoictic cells and, controlled by these regulatory sequences, a sequence coding for a protein or RNA with a therapeutic property when introduced into hemopoietic cells. Preferred examples of 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 .alpha.1-antitrypsin gene and the human multidrug resistance gene I (MDRI). The invention provides for effective gene therapy with PHSC of primates, in particular humans. | | US Patent Website: | Click Here for Full Text of Patent | | Title Number: | US6312957 | | Application Number: | US1999000326032 | | Date Filed: | 04/06/1999 | | Date Published: | 06/11/2001 | | Assignee: | Introgene B.V. Netherlands |
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