genetic modification of strain cells hematopoietic repopulation of primates.

摘要

This invention relates to a change GENETICS pluripotent hematopoietic CELL STRAINS OF PRIMATES (P PHSC) by transducing P - PHSC ASSOCIATED WITH A VIRUS adenoviruses (AAV) recombined. Recombined AAV genome includes a sequence of DNA flanked by Inverted Terminal Repeats (ITR) of the VPA. The DNA sequence typically includes normally regulatory sequences that are functional hematopoietic cells AND REGULATED BY THESE SEQUENCE regulatory sequences, encoding a protein or RNA, and having a therapeutic property when introduced hematopoietic cells. GENE glucocerebrosidase HUMAN lysosomal, a globin gene TO BE battery GENES BE - GLOBIN HUMAN, encoding a DNA sequence an RNA or protein having antiviral activity, GENE AL 1 - ANTITRIPSINO AND GENE I HUMAN multidrug RESISTANCE (MDRI) constitute preferred examples of DNA sequences. This invention relates also to a gene therapy EFFICIENT pluripotent hematopoietic cell STRAINS OF PRIMATES, AND IN PARTICULAR HUMAN BEINGS.