Methods for treating muscular dystrophy with bone marrow cells

A - Human Necessities – 61 – K

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A61K 35/28 (2006.01) A61K 48/00 (2006.01) A61P 21/00 (2006.01) C12N 5/06 (2006.01) C12N 5/10 (2006.01) A61K 35/12 (2006.01)

Patent

CA 2386536

Methods for treating muscle diseases via bone marrow transplantation of either allogeneic cells or autologous cells engineered to express dystrophin or other gene products affected in muscle diseases are disclosed. Bone marrow cells and bone marrow SP cells (a highly purified population of hematopoietic stem cells) can be used in the methods. Muscle diseases include muscular dystrophies, such as Duchenne muscular dystrophy, Becker muscular dystrophy and limb-girdle muscular dystrophies.

L'invention concerne des méthodes de traitement de maladies musculaires par transplantation soit de cellules allogéniques, soit de cellules autologues de la moelle osseuse, modifiées de sorte qu'elles expriment la dystrophine ou d'autres produits géniques atteints dans les maladies musculaires. Dans lesdites méthodes, on peut utiliser les cellules de la moelle osseuse et les cellules SP de la moelle osseuse (une population hautement purifiée de cellules souches hématopoïétiques). Les maladies musculaires englobent les dystrophies musculaires et notamment la dystrophie musculaire de Duchenne, la dystrophie musculaire de Becker et les myopathies des ceintures.

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