C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 5/0789 (2010.01) A61K 35/14 (2006.01) A61K 35/28 (2006.01) A61P 27/02 (2006.01) C12N 5/02 (2006.01) C12N 5/10 (2006.01) A61K 35/12 (2006.01) A61K 48/00 (2006.01)
Patent
CA 2493122
Isolated, mammalian, bone marrow-derived, lineage negative hematopoietic stem cell populations (Lin HSC) contain endothelial progenitor cells (EPC) capable of forming retinal blood vessels. At least about 50% of the cells in the isolated Lin HSC population include cell surface markers for CD31 and c-kit. Up to about 8% of the cells can include the Sca-1 cell marker, and up to about 4% of the cells can include the Flk-1/KDR marker. The isolated Lin HSC populations of the present invention are useful for treatment of ocular vascular diseases. The isolated Lin HSC populations that have been transfected with therapeutically useful genes are also provided, which are useful for delivering genes to the eye for cell-based gene therapy.
L'invention concerne des populations de cellules souches hématopoïétiques négatives de lignées, dérivées de la moelle osseuse, mammifères, isolées (Lin?-¿ HSC), contenant des cellules progénitrices endothéliales (EPC) capables de former des vaisseaux sanguins rétiniens. Au moins 50 % environ des cellules de la population Lin?-¿ HSC isolée comprennent des marqueurs de surface de CD31 et c-kit. Jusqu'à 8 % environ des cellules peuvent comprendre le marqueur Sca-1 et jusqu'à 4 % environ le marqueur Flk-1/KDR. Les populations Lin?-¿ HSC isolées de la présente invention sont utilisées pour le traitement de maladies vasculaires oculaires. L'invention concerne également des populations Lin?-¿ HSC isolées transfectées avec des gènes utiles sur le plan thérapeutique, permettant d'introduire des gènes dans l'oeil lors d'une thérapie génique à base de cellules.
Dasilva Karen
Friedlander Martin
Otani Atsushi
Cassan Maclean
The Scripps Research Institute
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