C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/12 (2006.01) A61K 48/00 (2006.01) C07K 14/72 (2006.01) C12N 9/64 (2006.01) C12N 15/57 (2006.01) C12N 15/67 (2006.01) C12N 15/85 (2006.01) C12Q 1/68 (2006.01)
Patent
CA 2362970
The invention relates to the use of a nucleic acid contruct comprising at least one hormone responsive element and a transgene for preparing an agent for gene transfer. It further relates to particular nucleic acid contructs comprising at least one hormone responsive element and a transgene, wherein one of said at least one hormone responsive elements is not functionally linked to the transgene, vectors comprising such nucleic acid contructs and compositions of matter comprising such nucleic acid constructs wherein the hormone responsive elements of the constructs are coupled to a hormone-hormone receptor complex. The nucleic acid constructs, plasmids, and compositions of matter of the invention have applications in gene therapy, particularly in the treatment of human blood clotting disorders, such as hemophilia. They may also be used to up- or down-regulate target genes and for the delivery of vaccines.
L'invention concerne l'utilisation d'une construction d'acides nucléiques comprenant au moins un élément hormono-sensible, ainsi qu'un transgène, servant à la préparation d'un transfert génique. Elle concerne en outre des constructions d'acides nucléiques en particulier comprenant au moins un élément hormono-sensible, ainsi qu'un transgène, l'un au moins de ces éléments sensibles n'étant pas lié de manière fonctionnelle au transgène; elle concerne également des vecteurs comprenant de telles constructions d'acides nucléiques ainsi que des compositions de matière comprenant de telles constructions, dans lesquelles les éléments des constructions, hormono-sensibles, sont couplés à un couple hormone-récepteur hormonal. On peut utiliser ces constructions d'acides nucléiques, des plasmides et compositions de matière de l'invention en thérapie génique, notamment dans le traitement des troubles de la coagulation sanguine humaine, tels que l'hémophilie. On peut les utiliser également pour réguler de manière positive ou négative les gènes cibles et pour l'apport de vaccins.
Bereskin & Parr Llp/s.e.n.c.r.l.,s.r.l.
Octagene Gmbh
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