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    • 10. 发明专利
    • PORCINE ANIMALS LACKING ANY EXPRESSION OF FUNCTIONAL α-1, 3-GALACTOSYLTRANSFERASE
    • 挥发性动物缺乏功能性α-1,3-肌动蛋白转移酶的任何表达
    • JP2011015697A
    • 2011-01-27
    • JP2010204963
    • 2010-09-13
    • Revivicor Incレビビコア, インコーポレイテッド
    • PHELPS CAROL J
    • A01K67/027A61K35/39A61L27/00A61P37/06C12N5/00C12N5/06C12N5/10C12N9/10C12N9/40C12N15/02C12N15/09C12N15/85
    • A01K67/0276A01K2217/075A01K2227/108A01K2267/02A01K2267/025C12N9/2465C12N15/8509
    • PROBLEM TO BE SOLVED: To provide viable pigs which lack any expression of functional α-1,3-GT; to provide procine cells, tissues and organs, which lack any expression of functional α-1,3-GT, for use in xenotransplantation or other biomedical applications; and to provide a method to select and screen for porcine cells, which lack galactose α-1,3-galactose epitopes on the cell surface.SOLUTION: This invention is the production of the first live pigs lacking any functional expression of α-1,3-galactosyltransferase. The invention provides the complete inactivation of both alleles of the α-1,3-galactosyltransferase gene in pigs, thus overcoming longstanding hurdle and making xenotransplantation a reality. Eliminating the expression of this gene, resulting in a lack of galactose α-1,3-galactose epitopes on the cell surface. This represents the first and major step in eliminating hyperacute rejection in pig-to-human xenotransplantation therapy. The invention also provides organs, tissues, and cells derived from such porcine animals, which are useful for xenotransplantation.
    • 要解决的问题:提供缺乏功能性α-1,3-GT表达的活猪; 提供缺乏用于异种移植或其他生物医学应用的功能性α-1,3-GT的任何表达的原代细胞,组织和器官; 并提供了选择和筛选细胞表面缺乏半乳糖α-1,3-半乳糖表位的猪细胞的方法。本发明是生产缺乏α-1的任何功能性表达的第一只活猪, 3 - 半乳糖转移酶。 本发明提供了猪的α-1,3-半乳糖基转移酶基因的两个等位基因的完全失活,从而克服了长期的障碍并使异种移植成为现实。 消除该基因的表达,导致细胞表面缺乏半乳糖α-1,3-半乳糖表位。 这是消除猪与人类异种移植治疗中的超急性排斥反应的第一个重要步骤。 本发明还提供了来自这样的猪动物的器官,组织和细胞,其可用于异种移植。