发明名称 METHODS AND COMPOSITIONS FOR TREATING HUNTINGTON'S DISEASE
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.
申请公布号 US2017035839(A1) 申请公布日期 2017.02.09
申请号 US201615294062 申请日期 2016.10.14
申请人 Sangamo BioSciences, Inc. 发明人 Miller Jeffrey C.;Rebar Edward J.;Zhang H. Steve
分类号 A61K38/17;C12N15/90;C07K14/47 主分类号 A61K38/17
代理机构 代理人
主权项 1. A method of binding a zinc finger protein to a target site in an Htt gene, the target site comprising the sequence CAGCAG, CTGCTG, GCAGCA, GCTGCT, AGC, CAG, CTG, GCA, GCT or AAG in a cell, the method comprising providing to the cell a non-naturally occurring zinc finger protein comprising 4, 5 or 6 zinc finger domains ordered F1 to F4, F1 to F5 or F1 to F6, respectively, wherein the zinc finger protein binds to the Htt gene and comprises one- or two-finger domains at positions F2 or F2 and F3, the two-finger domains consisting of the recognition helix regions sequences in F2 and F3 as shown in a single row of Table 2C and further wherein: (i) F2 and F3 of the zinc finger protein comprise the recognition helix regions shown in SEQ ID NO:161, 162, 163, 164, 165, 166 or 167 to bind to the target sequence CAGCAG; (ii) F2 and F3 of the zinc finger protein comprise the recognition helix regions shown in SEQ ID NO: 168 to bind to the target sequence CTGCTG; (iii) F2 and F3 of the zinc finger protein comprise the recognition helix regions shown in SEQ ID NO:169, 170, 171 and 172 to bind to the target sequence GCAGCA; (iv) F2 and F3 of the zinc finger protein comprise the recognition helix regions shown in SEQ ID NO: 173 and 174 to bind to the target sequence GCTGCT; (v) F2 comprises the recognition helix region shown in SEQ ID NO:175 to bind to the target sequence AGC; (vi) F2 comprises the recognition helix region shown in SEQ ID NO: 176 or 177 to bind to the target sequence CAG; (vii) F2 comprises the recognition helix region shown in SEQ ID NO:42 or 148 to bind to the target sequence CTG; (viii) F2 comprises the recognition helix region shown in SEQ ID NO:6, 18 or 178 to bind to the target sequence GCA; (ix) F2 comprises the recognition helix region shown in SEQ ID NO:31 to bind to the target sequence GCT; and (x) F2 comprises the recognition helix region shown in SEQ ID NO:65 to bind to the target sequence AAG.
地址 Richmond CA US