Therapeutic applications of thrombomodulin gene via viral...

A - Human Necessities – 61 – K

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A61K 48/00 (2006.01) C12N 15/86 (2006.01) C12N 15/861 (2006.01)

Patent

CA 2515916

The present invention relates to methods and compositions for treating thrombotic diseases using gene delivery technologies. In one embodiment, a therapeutically effective amount of a TM gene product is expressed in a TM- deficient mammal using a viral or non viral vector. In another embodiment, the vector-mediated in vivo TM gene expression is used for the treatment of atherosclerotic cardiovascular disease, pulmonary hypertension, acute inflammatory diseases, end-stage renal failure disease, or Alzheimer disease. In yet another embodiment, a vector carrying a TM inhibitory polynucleotide in which the vector is introduced into a mammal to reduce the TM activity or TM gene expression in vivo.

La présente invention se rapporte à des méthodes et à des compositions de traitement de maladies thrombotiques utilisant des technologies d'administration de gènes. Dans un mode de réalisation, une quantité thérapeutiquement efficace d'un produit génétique TM est exprimée chez un mammifère à carence de TM utilisant un vecteur viral ou non viral. Dans un autre mode de réalisation, l'expression génique TM in vivo induite par vecteur est utilisée dans le traitement de l'athérosclérose, l'hypertension pulmonaire, les maladies inflammatoires aiguës, l'insuffisance rénale en phase terminale, ou la maladie d'Alzheimer. Dans un autre mode de réalisation, un vecteur portant un polynucléotide inhibiteur de TM, dans lequel le vecteur est introduit dans un mammifère, réduit l'activité de TM ou l'expression génétique de TM in vivo.

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