| Title of Invention | "VECTOR ENCODING HUMAN GLOBIN GENE AND USE THEREOF IN TREATMENT OF HEMOGLOBINOPATHIES". |
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| Abstract | Recombinant lentiviral vectors having a region encoding a functional globin gene; and large portions of the beta-globin locus control regions which include DNase I hypersensitive sites HS2, HS3 and HS4 provides expression of beta-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used in treatment of hemoglobinopathies, including beta-thalessemia and sickle-cell disease. For example, hematopoietic progenitor or stem cells may be transformed ex vivo and then restored to the patient. Selection processes may be used to increase the percentage of transformed cells in the returned population. For example, a selection marker which makes transformed cells more drug resistant than un-transformed cells allows selection by treatment of the cells with the corresponding drug. (FIG. - 1) |
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1628-kolnp-2003-granted-abstract.pdf
1628-kolnp-2003-granted-assignment.pdf
1628-kolnp-2003-granted-claims.pdf
1628-kolnp-2003-granted-correspondence.pdf
1628-kolnp-2003-granted-description (complete).pdf
1628-kolnp-2003-granted-drawings.pdf
1628-kolnp-2003-granted-examination report.pdf
1628-kolnp-2003-granted-form 1.pdf
1628-kolnp-2003-granted-form 13.pdf
1628-kolnp-2003-granted-form 18.pdf
1628-kolnp-2003-granted-form 3.pdf
1628-kolnp-2003-granted-form 5.pdf
1628-kolnp-2003-granted-gpa.pdf
1628-kolnp-2003-granted-letter patent.pdf
1628-kolnp-2003-granted-reply to examination report.pdf
1628-kolnp-2003-granted-specification.pdf
| Patent Number | 219556 | |||||||||||||||
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| Indian Patent Application Number | 01628/KOLNP/2003 | |||||||||||||||
| PG Journal Number | 19/200 | |||||||||||||||
| Publication Date | 09-May-2008 | |||||||||||||||
| Grant Date | 07-May-2008 | |||||||||||||||
| Date of Filing | 16-Dec-2003 | |||||||||||||||
| Name of Patentee | SLOAN KETTERING INSTITUTE FOR CANCER RESEARCH . | |||||||||||||||
| Applicant Address | 1275YORK ASVENUEM NEW YORK NY10021 USA. | |||||||||||||||
Inventors:
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| PCT International Classification Number | G65 | |||||||||||||||
| PCT International Application Number | PCT/US02/20988 | |||||||||||||||
| PCT International Filing date | 2002-07-01 | |||||||||||||||
PCT Conventions:
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