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1.METHODS AND COMPOSITIONS FOR REGULATION OF ZINC FINGER PROTEIN EXPRESSION 审中-公开
标题翻译: 调节锌指蛋白表达的方法和组合物公开(公告)号:EP3119878A4
公开(公告)日:2017-08-16
申请号:EP15765802
申请日:2015-03-18
发明人: FROELICH STEVEN , GREGORY PHILIP D , ZHANG H STEVE
CPC分类号: C12N15/63 , C12N15/86 , C12N2830/60
摘要: The present disclosure is in the field of genome engineering, particularly regulating targeted modification of the genome.
摘要翻译: 本公开内容处于基因组工程领域。
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公开(公告)号:EP2820159A4
公开(公告)日:2015-08-26
申请号:EP13755872
申请日:2013-02-28
发明人: MILLER JEFFREY C , REBAR EDWARD J , ZHANG H STEVE
IPC分类号: C12Q1/68
CPC分类号: A61K38/1709 , C07K14/4703 , C07K2319/00 , C07K2319/70 , C07K2319/71 , C07K2319/81 , C12N15/907
摘要: Disclosed herein are compositions comprising non-naturally occurring zinc finger domains, fusion proteins comprising these zinc finger domains, polynucleotides encoding these proteins, cells expressing these proteins and pharmaceutical compositions comprising these proteins or polynucleotides as well as methods of modifying an Htt gene using these compositions for treating or preventing Huntington's Disease.
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3.METHODS AND COMPOSITIONS FOR TREATING TRINUCLEOTIDE REPEAT DISORDERS 审中-公开
标题翻译: 方法和组合物,用于治疗遗传性三核苷酸重复障碍公开(公告)号:EP2461819A4
公开(公告)日:2013-07-31
申请号:EP10806737
申请日:2010-07-28
发明人: GREGORY PHILIP D , REBAR EDWARD J , ZHANG H STEVE
CPC分类号: A61K38/1709 , A61K38/00 , A61K48/00 , C07K14/47 , C07K2319/00 , C07K2319/71 , C07K2319/81 , C12N15/907
摘要: Disclosed herein are methods and compositions for treating trinucleotide repeat disorders.
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公开(公告)号:EP3068905A4
公开(公告)日:2017-07-05
申请号:EP14860665
申请日:2014-11-11
发明人: ZHANG H STEVE
IPC分类号: C12Q1/68
CPC分类号: A61K38/465 , A61K9/0085 , A61K38/08 , A61K38/10 , A61K38/164 , A61K48/00 , C12N7/00 , C12N15/113 , C12N2310/10 , C12N2310/20 , C12N2320/34 , C12N2750/14143 , C12Y301/00
摘要: Disclosed herein are methods and compositions for diagnosing, treating or preventing Huntington's Disease.
摘要翻译: 公开了用于诊断,治疗亨廷顿氏病的方法和组合物。
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公开(公告)号:EP2997146A4
公开(公告)日:2017-04-26
申请号:EP14797155
申请日:2014-05-15
发明人: CONWAY ANTHONY , COST GREGORY J , DEKELVER RUSSELL , REBAR EDWARD J , REIK ANDREAS , URNOV FYODOR , WANG JIANBIN , ZHANG H STEVE
IPC分类号: C12N15/11
CPC分类号: C12N15/907 , C07K2/00 , C07K2319/81 , C12N9/22 , C12N15/11 , C12N15/90 , C12N15/902 , C12N2320/30 , C12Y301/21004
摘要: Methods and compositions comprising single guide RNAs targeted to an endogenous mammalian β globin gene (HBB), gamma globin gene (HBG1), B-cell lymphoma/leukemia 11A (BCL11A) gene, Kruppel-like factor 1 (KLF1) gene, CCR5 gene, CXCR4 gene, PPP1R12C (AAVS1) gene, hypoxanthine phosphoribosyltransferase (HPRT) gene, albumin gene, Factor VIII gene, Factor IX gene, Leucine-rich repeat kinase 2 (LRRK2) gene, Hungtingin (Htt) gene, rhodopsin (RHO) gene, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, surfactant protein B gene (SFTPB), T-cell receptor alpha (TRAC) gene, T-cell receptor beta (TRBC) gene, programmed cell death 1 (PD1) gene, Cytotoxic T-Lymphocyte Antigen 4 (CTLA-4) gene, human leukocyte antigen (HLA) A gene, a LMP7 gene, a Transporter associated with Antigen Processing (TAP) gene, tapasin gene (TAPBP), a class II major histocompatibility complex transactivator (CIITA) gene, a dystrophin gene (DMD), a glucocorticoid receptor gene (GR), IL2RG gene or RFX5 for genetic alteration of cells are provided.
摘要翻译: 提供了细胞的遗传改变的方法和组合物。
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