Oligonucleotide compositions and their use to induce...

A - Human Necessities – 61 – K

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A61K 31/7105 (2006.01) A61P 7/00 (2006.01) C12N 15/11 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2445071

The present invention provides compositions comprising a 3'-OH, 5'-OH, chemically unmodified, synthetic phosphodiester nucleotide sequence selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, and SEQ ID NO: 4, and a pharmaceutically acceptable carrier, wherein the compositions are useful to induce differentiation of cells or to stimulate differentiation or production of pluripotent cells. The present invention provides methods of using these compositions to induce differentiation of pluripotent cells, including bone marrow derived cells, and to treat disease associated with insufficient differentiation of cells in animals and humans, including but not limited to leukemia, lymphoma, non-malignant blood disorders such as hemoglobinopathies, sickle cell disease, myelodysplastic syndrome, pancytopenia, anemia, thrombocytopenia and leukopenia.

L'invention concerne des compositions comprenant une séquence nucléotidique phosphodiester synthétique 3'-OH, 5'-OH, non modifiée chimiquement, sélectionnée dans le groupe constitué de SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3 et SEQ ID NO: 4, et un support pharmaceutiquement acceptable, ces compositions étant utiles pour induire la différentiation cellulaire ou pour stimuler la différentiation ou la production de cellules pluripotentielles. La présente invention porte également sur des procédés d'utilisation de ces compositions pour induire une différentiation de cellules pluripotentielles comportant des cellules dérivées de moelle osseuse, et pour traiter des maladies associées à l'insuffisance de différentiation cellulaire chez les animaux et les humains, incluant mais sans se limiter à la leucémie, les lymphomes, les troubles sanguins bénins tels que les hémoglobinopathies, la drépanocytose, le syndrome myélodysplasique, la pancytopénie, l'anémie, la thrombocytopénie et la leucopénie.

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