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2. 发明申请

WO2014159312A2 A NOVEL SINGLE CHAIN ANTIBODY REDUCED MUTANT ALPHA-1 ANTITRYPSIN AGGREGATION AND TOXICITY 审中-公开
标题翻译：新型单链抗体减少突变型ALPHA-1抗菌素聚集与毒性
公开(公告)号：WO2014159312A2
公开(公告)日：2014-10-02
申请号：PCT/US2014/022952
申请日：2014-03-11
申请人： UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED
发明人： BRANTLY, Mark, Louis , XIAO, Kai , LEVITES, Yona , WANG, Liqun , SONG, Sihong , ROUHANI, Farshid, N. , OSHINS, Regina, A.
CPC分类号： C07K16/38 , A61K2039/505 , C07K2317/622
摘要： The current invention is directed to a gene therapy comprising, introducing genes to a subject, wherein the genes encode fusion proteins comprising proteins or polypeptides that target the fusion proteins to proteasomal degradation pathways and antibodies or fragments of the antibodies against mutant or abnormal proteins that misfold and accumulate in the ER causing the disease. The current invention is also directed to gene therapy, comprising introducing genes to a subject, wherein the genes encode fusion proteins comprising FKBP12 and antibodies or a fragment of the antibodies against mutant or abnormal proteins that misfold and accumulate in the ER causing the disease. The current invention is further directed to a gene therapy for alpha- 1 antitrypsin deficiency, comprising introducing genes to a human in need of the gene therapy, wherein the genes encode fusion proteins comprising FKBP12 and scFv portion of a monoclonal antibody against alpha- 1 antitrypsin.
摘要翻译：本发明涉及基因治疗，其包括将基因导入受试者，其中所述基因编码融合蛋白，所述融合蛋白包含靶向所述融合蛋白的蛋白质或多肽以及蛋白酶体降解途径，以及针对错误折叠的突变或异常蛋白的抗体或抗体片段并累积在引起疾病的ER中。本发明还涉及基因治疗，其包括将基因导入受试者，其中所述基因编码包含FKBP12和抗体的融合蛋白或针对突变体或异常蛋白质的抗体片段，其错误地折叠并累积在引起疾病的ER中。本发明进一步针对α-1抗胰蛋白酶缺乏症的基因疗法，其包括将基因导入需要进行基因治疗的人，其中所述基因编码包含抗α-1抗胰蛋白酶的单克隆抗体的FKBP12和scFv部分的融合蛋白。

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