Gene therapy agent for Haemophilia B and its preparation method

Details Gene therapy agent for Haemophilia B and its preparation method Gene therapy agent for Haemophilia B and its preparation method

2008-04-22

2008-04-22

Woitach, Joseph (Department: 1633)

Drug, bio-affecting and body treating compositions

Designated organic active ingredient containing

Carbohydrate doai

C435S320100, C424S093100, C536S023100

Reexamination Certificate

active

07361639

ABSTRACT:
The invention relates to site-specific integrating expression vectors for therapy Hemophilia B and to methods of preparing them. A vector of the invention contains a human Factor IX gene in a vector constructed using as a chromosome targeting sequence a polynucleotide without any important physiological function-related gene homologous to DNA on the short arms of human group D and human group G chromosomes. The vector of the invention provides high stability of Factor IX expression, high expression efficiency, no immunogenicity and safety in use.

REFERENCES:
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Eck, et al. (1996) Goodman & Gilman's The Pharmacological Basis of Therapeutics, 9th Ed., McGraw-Hill, New York, NY., pp. 77-101.
Walsh (2003) Gene Therapy, 10: 999-1003.
Garret and Grisham (1995) Biochemistry, Harcourt Brace College Publishers, New York, NY., pp. 916-917.
Yanez, et al. (1998) Gene Therapy, 5: 149-159.

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