发明名称 Pancreatic Islets of Transgenic LEA29Y Animals for Treating Diabetes
摘要 The present invention relates to methods of treating diabetes in a human subject comprising the use of pancreatic islets or of embryonic pancreatic tissue of a transgenic animal, wherein said transgenic animal contains a polynucleotide sequence encoding a CTLA4 peptide-immunoglobulin fusion, preferably LEA29Y, and expresses said CTLA4 peptide-immunoglobulin fusion in a tissue-specific manner in pancreatic islets.
申请公布号 US2017042942(A1) 申请公布日期 2017.02.16
申请号 US201615336350 申请日期 2016.10.27
申请人 MWM Biomodels GmbH 发明人 WOLF Eckhard;KLYMIUK Nikolai;WOLF-VAN BUERCK Lelia;SEIßLER Jochen
分类号 A61K35/39;A01K67/027 主分类号 A61K35/39
代理机构 代理人
主权项 1. A method of treating diabetes in a human subject, comprising the steps of: isolating pancreatic islets of a transgenic animal and administering said isolated pancreatic islets of the transgenic animal into a human subject in need thereof, wherein said isolated pancreatic islets of the transgenic animal have the ability to normalize blood glucose levels in the human subject into which the islets are administered, wherein said transgenic animal is a transgenic animal whose genome comprises a recombinant nucleic acid comprising a polynucleotide sequence encoding a CTLA4 peptide fused to an immunoglobulin (“CTLA4 peptide-immunoglobulin fusion”) wherein said polynucleotide sequence is operably linked to an insulin promoter that results in expression of the CTLA4 peptide-immunoglobulin fusion, wherein said animal expresses the CTLA4 peptide-immunoglobulin fusion, wherein said expression is stable in offspring of said transgenic animal, wherein said animal exhibits, as a result of the expression of said CTLA4 peptide-immunoglobulin fusion, tissue-specific expression of the CTLA4 peptide-immunoglobulin fusion in pancreatic islets; and wherein after xenotransplantation of said transgenic pancreatic islets of said animal into a human subject, said transgenic pancreatic islets are protected from rejection by mature human immune system cells, normalize blood glucose levels in the human subject, and prevent re-occurrence of hyperglycemia.
地址 Tiefenbach DE