Genetic modification of primate hemopoietic repopulating stem cells
US6312957A · kind A · utility
Assignee
Inventors
Key dates
| Filing date | Jun 4, 1999 |
| Grant date | Nov 6, 2001 |
| Priority date | — |
| Expiry date | Jun 4, 2019 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.