Compositions and methods for gene therapy to treat disease

Details Compositions and methods for gene therapy to treat disease Compositions and methods for gene therapy to treat disease

A - Human Necessities

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Patent

CA 2188675

Method of gene therapy for the treatment of a variety of genetic defects or diseases comprising the steps of either introducing transformed cells into an organism such as a human or the in vivo transforma- tion of cells. The transformed cells contain a nucleic acid vector which will produce the protein neces- sary to correct the deficiency or disease. Examples of diseases which can be treated this way include diabetes mellitus, Parkinson's disease, cardiovascu- lar disease, hypercholesterolemia, hypertension, ane- mia, thrombosis, growth disorders, metabolic disor- ders such as PKU and serum protein disorders, such as hemophilia A.

Procédé de thérapie génique destiné au traitement de toute une série d'anomalies ou maladies génétiques, qui consiste soit à introduire des cellules transformées dans un organisme tel qu'un être humain, soit à transformer les cellules in vivo. Les cellules transformées contiennent un vecteur d'acide nucléique qui produira la protéine nécessaire pour corriger l'anomalie ou la maladie. Parmi les maladies qui peuvent être traitées de cette manière, on trouve le diabète sucré, la maladie de Parkinson, les maladies cardio-vasculaires, l'hypercholestérolémie, l'hypertension, l'anémie, la thrombose, les troubles de la croissance, les troubles métaboliques tels que la polycétonurie et les troubles des protéines sériques, tels que l'hémophilie A.

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