JP2013544077A5 - - Google Patents
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- JP2013544077A5 JP2013544077A5 JP2013533982A JP2013533982A JP2013544077A5 JP 2013544077 A5 JP2013544077 A5 JP 2013544077A5 JP 2013533982 A JP2013533982 A JP 2013533982A JP 2013533982 A JP2013533982 A JP 2013533982A JP 2013544077 A5 JP2013544077 A5 JP 2013544077A5
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18.前記対象の選択を、胚、胎児、新生児、幼児、若年者、成人からなるグループの中から行なう、8〜17のいずれか1項に記載の方法。
Claims (14)
- 改変したジンクフィンガータンパク質DNA結合ドメインを含むタンパク質であって、
そのDNA結合ドメインが、N末端からC末端に向けてF1〜F4またはF1〜F5の順番で並んだ4つまたは5つのジンクフィンガー認識領域を含み、
(i)DNA結合ドメインが、5つのジンクフィンガー認識領域を含んでいるときには、F1〜F5は、以下のアミノ酸配列:
F1:QSGDLTR(配列番号4)
F2:RSDVLSE(配列番号5)
F3:DRSNRIK(配列番号6)
F4:RSDNLSE(配列番号7)
F5:QNATRIN(配列番号8)を含み;
(ii)DNA結合ドメインが、4つのジンクフィンガー認識領域を含んでいるときには、F1〜F4は、以下のアミノ酸配列:
F1:RSDSLSV(配列番号10)
F2:TSGHLSR(配列番号11)
F3:RSDHLSQ(配列番号12)
F4:HASTRHC(配列番号13)を含む、タンパク質。 - 野生型もしくは改変された開裂ドメインまたは開裂半ドメインをさらに含む、請求項1に記載のタンパク質。
- 請求項1または2に記載のタンパク質をコードするポリヌクレオチド。
- 請求項1または2に記載のタンパク質、または請求項3に記載のポリヌクレオチドを含む、単離された細胞。
- 対象の血友病Bを治療するための、肝細胞を含む医薬組成物であって、
機能的なIX因子(FIX)タンパク質をコードする配列が、少なくとも1つのジンクフィンガーヌクレアーゼを用いて該細胞のゲノムに組み込まれ、該ジンクフィンガーヌクレアーゼは、請求項1又は2に記載のタンパク質であるか、又は請求項3に記載のポリヌクレオチドによってコードされる、医薬組成物。 - 前記配列が内在性遺伝子に組み込まれている、請求項5に記載の医薬組成物。
- 前記内在性遺伝子が、FIX遺伝子およびセーフハーバー遺伝子からなる群より選択される、請求項5または6に記載の医薬組成物。
- 前記細胞が肝細胞であり、前記配列が、傷のない動物の肝臓に静脈内投与することによって、または腹腔内投与することによって、または肝臓の柔組織に直接注入することによって、または肝動脈に注入することによって、または胆管を通じて逆行注入することによって、その細胞に送達される、請求項5〜7のいずれか1項に記載の医薬組成物。
- 対象の部分的肝除去が更に実施され、および/またはガンマ線照射、UV照射、トリチウム標識したヌクレオチド、シスプラチン、エトポシド、ヒドロキシ尿素、アフィジコリン、プレドニゾロン、アデノウイルス、ならびにこれらの組み合わせからなる群より選択される少なくとも1種類の二次剤が対象に更に投与される、請求項5〜8のいずれか1項に記載の医薬組成物。
- 請求項5〜9のいずれか1項に記載の医薬組成物であって、前記細胞が単離された細胞である、医薬組成物。
- 前記対象が、胚、胎児、新生児、幼児、若年者、成人からなる群より選択される、請求項5〜10のいずれか1項に記載の医薬組成物。
- 前記配列、前記ジンクフィンガーヌクレアーゼ、そのジンクフィンガーヌクレアーゼをコードするポリヌクレオチド、これらの組み合わせのいずれかが、前記細胞の表面受容体に特異的に結合するホーミング剤と組み合わされる、請求項5〜11のいずれか1項に記載の医薬組成物。
- 前記ホーミング剤が、ガラクトースを含むか、AAVコートタンパク質とガラクトースのハイブリッドを含む、請求項12に記載の医薬組成物。
- 前記細胞が、ヒト細胞、非ヒト霊長類細胞、齧歯目細胞、ウサギ目細胞、食肉目細胞、偶蹄類細胞と、幹細胞である胚性幹細胞、造血幹細胞、人工多能性幹細胞、肝細胞、および肝幹細胞からなる群より選択される、請求項8〜13のいずれか1項に記載の医薬組成物。
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
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US39233310P | 2010-10-12 | 2010-10-12 | |
US61/392,333 | 2010-10-12 | ||
PCT/US2011/056020 WO2012051343A1 (en) | 2010-10-12 | 2011-10-12 | Methods and compositions for treating hemophilia b |
Publications (3)
Publication Number | Publication Date |
---|---|
JP2013544077A JP2013544077A (ja) | 2013-12-12 |
JP2013544077A5 true JP2013544077A5 (ja) | 2016-01-07 |
JP6018069B2 JP6018069B2 (ja) | 2016-11-02 |
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Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
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JP2013533982A Active JP6018069B2 (ja) | 2010-10-12 | 2011-10-12 | 血友病bを治療する方法及び組成物 |
Country Status (12)
Country | Link |
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US (2) | US9175280B2 (ja) |
EP (1) | EP2627665B1 (ja) |
JP (1) | JP6018069B2 (ja) |
KR (1) | KR101930711B1 (ja) |
CN (1) | CN103270046B (ja) |
AU (1) | AU2011316575B2 (ja) |
CA (1) | CA2814143C (ja) |
ES (1) | ES2562421T3 (ja) |
HK (1) | HK1185623A1 (ja) |
RU (1) | RU2608643C2 (ja) |
WO (1) | WO2012051343A1 (ja) |
ZA (1) | ZA201302625B (ja) |
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AU2008305567B2 (en) | 2007-09-27 | 2014-04-10 | Sangamo Therapeutics, Inc. | Rapid in vivo identification of biologically active nucleases |
EP2281050B1 (en) | 2008-04-14 | 2014-04-02 | Sangamo BioSciences, Inc. | Linear donor constructs for targeted integration |
EP2352369B1 (en) | 2008-12-04 | 2017-04-26 | Sangamo BioSciences, Inc. | Genome editing in rats using zinc-finger nucleases |
EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
WO2010117464A1 (en) | 2009-04-09 | 2010-10-14 | Sangamo Biosciences, Inc. | Targeted integration into stem cells |
US20120192298A1 (en) * | 2009-07-24 | 2012-07-26 | Sigma Aldrich Co. Llc | Method for genome editing |
PT2534173T (pt) | 2010-02-08 | 2019-10-31 | Sangamo Therapeutics Inc | Semidomínios de clivagem manipulados |
WO2011146121A1 (en) | 2010-05-17 | 2011-11-24 | Sangamo Biosciences, Inc. | Novel dna-binding proteins and uses thereof |
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