Genetically modified cd34-negative adherently growing stem...

Details Genetically modified cd34-negative adherently growing stem... Genetically modified cd34-negative adherently growing stem...

C - Chemistry, Metallurgy

12

N

C12N 5/10 (2006.01) A61K 35/14 (2006.01) A61K 35/12 (2006.01) A61K 48/00 (2006.01) C12N 5/06 (2006.01)

Patent

CA 2338541

The invention relates to the use of genetically modified earliest haematopoietic and mesenchymal stem cells (which are negative for the expression of the CD34surface molecule) in individual gene therapy of mono- or oligogenetic diseases, respectively, or in cell therapy. For this purpose, endogenous CD34-negative adherently growing stem cell cultures are established from the peripheral blood of the patient and efficiently transfected or infected, respectively, with gene constructs. The gene products of said genes shall substitute defective or missing proteins or factors, respectively, in the patient organism on a long term basis. After expansion said endogenous stem cells may also be used for cell therapy (organ replacement therapy).

L'invention concerne l'utilisation de cellules souches génétiquement modifiées, hématopoïétiques et mésenchymateuses précoces (négatives pour l'expression de la molécule de surface CD34) en thérapie génique individuelle de maladies monogénétiques et oligogénétiques ou en thérapie cellulaire. A cet effet, des cultures de cellules souches autologues croissantes par adhésion, CD34-négatives, issues du sang périphérique du patient, sont appliquées puis efficacement transfectées ou infectées avec des constructions géniques. Les produits géniques de ces gènes doivent pallier les défauts ou substituer les protéines ou facteurs manquants de l'organisme du patient. Après expansion, les cellules souches autologues peuvent également être utilisées en thérapie cellulaire (thérapie de remplacement des organes).

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