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Tommande Un Cauri Modul Pentini TOMMANDE US009877988B2UN CAURIMODUL PENTINI (12 ) United States Patent ( 10 ) Patent No. : US 9 ,877 , 988 B2 Rebar (45 ) Date of Patent: * Jan . 30 , 2018 ( 54 ) METHOD OF TREATING LYSOSOMAL 6 , 479 ,626 B1 11/ 2002 Kim et al. STORAGE DISEASES USING NUCLEASES 6 , 503 , 717 B2 1 / 2003 Case et al . 6 , 534 ,261 B1 3 / 2003 Cox , III et al. AND A TRANSGENE 6 , 599 , 692 B1 7 / 2003 Case et al. 6 , 607, 882 B1 8 / 2003 Cox et al . (71 ) Applicant: Sangamo BioSciences , Inc ., Richmond, 6 , 689 ,558 B2 2 / 2004 Case 6 , 723 , 551 B2 4 /2004 Ding et al . CA (US ) 6 , 794 , 136 B1 9 /2004 Eisenberg et al . 6 , 824 , 978 B1 11/ 2004 Cox , III et al. ( 72 ) Inventor: Edward J . Rebar, Richmond , CA (US ) 6 , 903 , 185 B2 6 / 2005 Kim et al. 6 , 933 , 113 B2 8 / 2005 Case et al . ( 73 ) Assignee : Sangamo Therapeutics, Inc. , 6 , 979, 539 B2 12/ 2005 Cox , III et al. Richmond , CA (US ) 7 , 013 , 219 B2 3 /2006 Case et al. 7 , 030 , 215 B2 4 / 2006 Liu et al. 7 , 067 , 317 B2 6 / 2006 Rebar et al . ( * ) Notice : Subject to any disclaimer , the term of this 7 , 070, 934 B2 7 /2006 Cox , III et al. patent is extended or adjusted under 35 7 , 153 , 949 B2 12 / 2006 Kim et al. U . S . C . 154 ( b ) by 0 days . 7 , 163, 824 B2 1 / 2007 Cox , III et al. This patent is subject to a terminal dis 7 , 253 , 273 B2 8 /2007 Collinowood NNNNN7 ,262 , 054 B2 8 /2007 Jamieson et al. claimer . 7 ,361 , 635 B2 4 / 2008 Miller et al. 7 , 888 , 121 B2 2 / 2011 Umov et al . ( 21) Appl. No. : 13 /839 , 336 8 , 697 , 359 B1 4 / 2014 Zhang 8 , 771 , 945 B1 . 7 / 2014 Zhang 8 , 771 , 985 B2 * 7 / 2014 Cui et al. 435 /69 . 1 ( 22 ) Filed : Mar. 15 , 2013 8 ,795 , 965 B2 8 / 2014 Zhang 8 , 865 ,406 B2 10 /2014 Zhang et al. (65 ) Prior Publication Data 8 , 871 ,445 B2 10 / 2014 Cong et al . 8 , 889 , 356 B2 11/ 2014 Zhang US 2014 / 0017212 A1 Jan . 16 , 2014 8 ,889 ,418 B2 11 /2014 Zhang et al. 8 , 895 , 308 B1 11/ 2014 Zhang et al . Related U . S . Application Data (Continued ) ( 60 ) Provisional application No . 61/ 670 , 463 , filed on Jul . 11, 2012 , provisional application No . 61 / 704 ,072 , FOREIGN PATENT DOCUMENTS filed on Sep . 21, 2012 . GB 2338237 12 / 1999 WO WO 95/ 19431 7 / 1995 (51 ) Int . CI. AOIN 63 / 00 ( 2006 .01 ) ( Continued ) A61K 35 /28 ( 2015 .01 ) A61K 38 /46 ( 2006 . 01 ) OTHER PUBLICATIONS C12N 9 / 22 ( 2006 .01 ) High (Nature , 2005 , vol. 435 , p . 577 - 578) . * C12N 15 / 90 ( 2006 .01 ) Deonarain (1998 , Expert Opin . Ther. Pat ., vol . 8 , pp . 53 -69 ). * A61K 48 /00 ( 2006 .01 ) Verma (Sep . 1997 , Nature , vol. 389 , pp . 239 - 242 ) . * (52 ) U . S . CI. Palu ( J . Biotechnol . , 1999 , vol. 68 , p . 1 - 13 ) . * CPC . .. .. .. A61K 35 / 28 ( 2013 .01 ) ; A61K 38 / 46 Luo (Nature Biotechnol. , 2000 , vol . 18 , p . 33 -37 ) . * ( 2013 .01 ) ; A61K 38 / 465 ( 2013 .01 ) ; A61K Pfeifer ( Annu . Rev . Genomics . Hum . Genet . 2001, vol. 2 , p . 48 / 005 ( 2013 . 01 ) ; C12N 9 / 22 ( 2013 .01 ) ; 177 - 211. * C12N 15 / 907 (2013 . 01 ) ; A61K 48 / 00 Bibikova (Mol . Cellular Biol ., 2001, vol. 21 , No . 1 , p . 289 - 297) . * (2013 . 01 ) ; CO7K 2319/ 81 ( 2013 .01 ) Edelstein ( Journal Gene Med. , 2004 , vol. 6 , p . 597 -602 ) . * (58 ) Field of Classification Search Porteus (Nature Biotechnology , Aug. 2005 , vol . 23 , No. 8 , p . USPC .. .. .. 435 /325 , 455 ; 424 / 93 .21 , 93 . 2 ; 514 /44 ; 967- 973 ) . * 800 / 8 , 18 , 21 (Continued ) See application file for complete search history . (56 ) References Cited Primary Examiner - Michael Wilson (74 ) Attorney, Agent, or Firm — Pasternak Patent Law ; U . S . PATENT DOCUMENTS Susan Abrahamson 5 , 356 , 802 A 10 / 1994 Chandrasegaran 5 , 436 , 150 A 7 / 1995 Chandrasegaran 5 ,487 , 994 A 1 / 1996 Chandrasegaran (57 ) ABSTRACT 5 ,789 , 538 A 8 / 1998 Rebar et al . 5 , 925 , 523 A 7 / 1999 Dove et al. Nucleases and methods of using these nucleases for insert 6 , 007 , 988 A 12 / 1999 Choo ing a sequence encoding a therapeutic protein such as an 6 ,013 , 453 A 1 / 2000 Choo enzyme into a cell , thereby providing proteins or cell thera 6 , 140 ,081 A 10 / 2000 Barbas 6 , 140 ,466 A 10 / 2000 Barbas, III et al. peutics for treatment and / or prevention of a lysosomal 6 , 200 ,759 B1 3 / 2001 Dove et al . storage disease . 6 , 242 , 568 B1 . 6 / 2001 Barbas et al . 6 , 410, 248 B1 6 / 2002 Greisman et al . 6 ,453 , 242 B1 9 / 2002 Eisenberg et al . 3 Claims, 9 Drawing Sheets US 9 ,877 Page, 2988 B2 (56 ) References Cited Morton (PNAS , Oct. 13 , 2006 , vol. 13 , No . 44 , p . 16370 - 16375 ) . * Doyon (Nature Biotech . , Jun . 2008, vol. 26 , No. 6 , p . 702 - 708 ). * U . S . PATENT DOCUMENTS Meng (Nature Biotechnology , Jun . 2008 , vol. 26 , No . 6 , p . 695 701 ) . * 8 ,906 ,616 B2 12 / 2014 Zhang et al . 8 . 932 . 814 B2 1 / 2015 Cong et al. 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