JP2018527013A5 - - Google Patents
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- JP2018527013A5 JP2018527013A5 JP2018515082A JP2018515082A JP2018527013A5 JP 2018527013 A5 JP2018527013 A5 JP 2018527013A5 JP 2018515082 A JP2018515082 A JP 2018515082A JP 2018515082 A JP2018515082 A JP 2018515082A JP 2018527013 A5 JP2018527013 A5 JP 2018527013A5
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- 150000001875 compounds Chemical class 0.000 claims 27
- 239000002773 nucleotide Substances 0.000 claims 11
- 125000003729 nucleotide group Chemical group 0.000 claims 9
- 229920000033 CRISPR Polymers 0.000 claims 5
- 238000010354 CRISPR gene editing Methods 0.000 claims 5
- 229920003013 deoxyribonucleic acid Polymers 0.000 claims 5
- 239000000178 monomer Substances 0.000 claims 5
- 201000010099 disease Diseases 0.000 claims 3
- 239000003937 drug carrier Substances 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 108010082319 CRISPR-Associated Protein 9 Proteins 0.000 claims 2
- IQFYYKKMVGJFEH-XLPZGREQSA-N DEOXYTHYMIDINE Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims 2
- 241001367079 Una Species 0.000 claims 2
- DRTQHJPVMGBUCF-UCVXFZOQSA-N Uridine Natural products O[C@H]1[C@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UCVXFZOQSA-N 0.000 claims 2
- 229940045145 Uridine Drugs 0.000 claims 2
- JCAQMQLAHNGVPY-UUOKFMHZSA-N [(2R,3S,4R,5R)-3,4-dihydroxy-5-(2,2,4-trioxo-1H-imidazo[4,5-c][1,2,6]thiadiazin-7-yl)oxolan-2-yl]methyl dihydrogen phosphate Chemical class O[C@@H]1[C@H](O)[C@@H](COP(O)(O)=O)O[C@H]1N1C(NS(=O)(=O)NC2=O)=C2N=C1 JCAQMQLAHNGVPY-UUOKFMHZSA-N 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 230000003612 virological Effects 0.000 claims 2
- ASUCSHXLTWZYBA-UMMCILCDSA-N 2-amino-8-bromo-9-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-3H-purin-6-one Chemical compound C1=2NC(N)=NC(=O)C=2N=C(Br)N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O ASUCSHXLTWZYBA-UMMCILCDSA-N 0.000 claims 1
- AGFIRQJZCNVMCW-UAKXSSHOSA-N 5-Bromouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(Br)=C1 AGFIRQJZCNVMCW-UAKXSSHOSA-N 0.000 claims 1
- 102200057990 ASGR2 G85R Human genes 0.000 claims 1
- 206010049460 Abasia Diseases 0.000 claims 1
- 102100007326 BIRC5 Human genes 0.000 claims 1
- 102100008201 CDK16 Human genes 0.000 claims 1
- 101700024634 CDK16 Proteins 0.000 claims 1
- 102000012605 Cystic Fibrosis Transmembrane Conductance Regulator Human genes 0.000 claims 1
- 108010079245 Cystic Fibrosis Transmembrane Conductance Regulator Proteins 0.000 claims 1
- 125000000824 D-ribofuranosyl group Chemical group [H]OC([H])([H])[C@@]1([H])OC([H])(*)[C@]([H])(O[H])[C@]1([H])O[H] 0.000 claims 1
- 102100019126 HBB Human genes 0.000 claims 1
- 108091005902 Hemoglobin subunit beta Proteins 0.000 claims 1
- 229920000460 Mitochondrial DNA Polymers 0.000 claims 1
- VQAYFKKCNSOZKM-IOSLPCCCSA-N N6-Methyladenosine Chemical class C1=NC=2C(NC)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O VQAYFKKCNSOZKM-IOSLPCCCSA-N 0.000 claims 1
- 229940052778 Neisseria meningitidis Drugs 0.000 claims 1
- 241000588650 Neisseria meningitidis Species 0.000 claims 1
- 102200084290 PRCD V30M Human genes 0.000 claims 1
- 102100008800 SOD1 Human genes 0.000 claims 1
- 102100019667 STAT3 Human genes 0.000 claims 1
- 108010017324 STAT3 Transcription Factor Proteins 0.000 claims 1
- 241000193998 Streptococcus pneumoniae Species 0.000 claims 1
- 229940031000 Streptococcus pneumoniae Drugs 0.000 claims 1
- 241000193996 Streptococcus pyogenes Species 0.000 claims 1
- 229940076156 Streptococcus pyogenes Drugs 0.000 claims 1
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 1
- 108010002687 Survivin Proteins 0.000 claims 1
- 229940104230 Thymidine Drugs 0.000 claims 1
- 229920001723 Trans-activating crRNA Polymers 0.000 claims 1
- HDZZVAMISRMYHH-LITAXDCLSA-N Tubercidin Chemical compound C1=CC=2C(N)=NC=NC=2N1[C@@H]1O[C@@H](CO)[C@H](O)[C@H]1O HDZZVAMISRMYHH-LITAXDCLSA-N 0.000 claims 1
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 1
- 125000003342 alkenyl group Chemical group 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000000304 alkynyl group Chemical group 0.000 claims 1
- 239000005547 deoxyribonucleotide Substances 0.000 claims 1
- 229910052736 halogen Inorganic materials 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- -1 propyl uridine Chemical compound 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
Claims (25)
- ゲノムDNAを標的とするガイド化合物であって、crRNAと結合している14〜24個の連続するモノマーの標的ガイド鎖を含み、ゲノムDNAのCRISPR遺伝子編集を導き、モノマーがUNAモノマーおよび核酸モノマーを含み、ガイド化合物が、ゲノムDNAのCRISPR遺伝子編集を導くことを標的とする塩基配列を含む、ガイド化合物。
- ガイド化合物が、ヒト遺伝子TTRに二本鎖切断を導き、標的ガイド鎖が、5’−UGCAUGGCCACAUUGAUGGC−3’(配列番号13)の16〜20個の連続するモノマーを含み、crRNAが、標的ガイド鎖の3’末端において結合している、請求項1に記載のガイド化合物。
- 配列番号32を含む、請求項2に記載のガイド化合物。
- ガイド化合物が、ヒト遺伝子TTRに二本鎖切断を導き、標的ガイド鎖が、5’−CACAUGCAUGGCCACAUUGA−3’(配列番号40)の16〜20個の連続するモノマーを含み、crRNAが、標的ガイド鎖の3’末端において結合している、請求項1に記載のガイド化合物。
- 配列番号61を含む、請求項4に記載のガイド化合物。
- crRNAが、5’−GUUUUAGAGCUAUGCU−3’(配列番号605)である、請求項1に記載のガイド化合物。
- 標的ガイド鎖の塩基配列が、ゲノムDNAから最大で3つのミスマッチを有する、請求項1に記載のガイド化合物。
- 1〜5個のUNAモノマーを含む、請求項1に記載のガイド化合物。
- 1以上の核酸モノマーが、2’−O−メチルリボヌクレオチド、2’−O−メチルプリンヌクレオチド、2’−デオキシ−2’−フルオロリボヌクレオチド、2’−デオキシ−2’−フルオロピリミジンヌクレオチド、2’−デオキシリボヌクレオチド、2’−デオキシプリンヌクレオチド、ユニバーサル塩基ヌクレオチド、5−C−メチルヌクレオチド、逆位デオキシ脱塩基モノマー残基、3’末端が安定化されたヌクレオチド、3’−グリセリルヌクレオチド、3’−逆位脱塩基ヌクレオチド、3’−逆位チミジン、ロックド核酸ヌクレオチド(LNA)、2’−O,4’−C−メチレン−(D−リボフラノシル)ヌクレオチド、2’−メトキシエトキシ(MOE)ヌクレオチド、2’−メチル−チオ−エチル、2’−デオキシ−2’−フルオロヌクレオチド、2’−O−メチルヌクレオチド、2’,4’−拘束2’−O−メトキシエチル(cMOE)、2’−O−エチル(cEt)、2’−アミノヌクレオチド、2’−O−アミノヌクレオチド、2’−C−アリルヌクレオチド、2’−O−アリルヌクレオチド、N6−メチルアデノシンヌクレオチド、修飾された塩基5−(3−アミノ)プロピルウリジンを有するヌクレオチド、修飾された塩基5−(2−メルカプト)エチルウリジンを有するヌクレオチド、修飾された塩基5−ブロモウリジンを有するヌクレオチド、修飾された塩基8−ブロモグアノシンを有するヌクレオチド、修飾された塩基7−デアザアデノシンを有するヌクレオチド、2’−O−アミノプロピル置換されたヌクレオチド、または2’−OH基を、2’−R、2’−OR、2’−ハロゲン、2’−SRもしくは2’−アミノに置換したヌクレオチド(ここでRは、H、アルキル、アルケニル、またはアルキニルであり得る)である、請求項1に記載のガイド化合物。
- ガイド化合物の各末端における、最後の3つのモノマーのうちの1つ以上が、ホスホロチオエート結合、キラルなホスホロチオエート結合またはホスホロジチオエート結合で連結されている、請求項1に記載のガイド化合物。
- TTR、BIRC5、CDK16、STAT3、CFTR、F9、KRASおよびCARから選択される遺伝子において、二本鎖切断を導く、請求項1に記載のガイド化合物。
- ゲノムDNAが標的疾患に関連する一塩基多型を含む、請求項1に記載のガイド化合物。
- 疾患関連アレルにおいて二本鎖切断を導く、請求項1に記載のガイド化合物。
- V30M TTR、G284R ColA1、L132Pケラチン12、R135Tケラチン12、G85R SOD1、G272V Tau、P301L Tau、V337M Tau、R406W Tau、Q39STOPベータ−グロビン、T8993G/C mtDNA、G719S EGFR、およびG12C Krasから選択される疾患関連アレルにおいて、二本鎖切断を導く、請求項1に記載のガイド化合物。
- 30〜300個の連続するモノマーを含む、請求項1に記載のガイド化合物。
- CRISPR遺伝子編集がCas9を用いる、請求項1に記載のガイド化合物。
- 疾患関連アレルにおいて、野生型としての同一のアレルよりも多くの二本鎖切断を導く、請求項1に記載のガイド化合物。
- tracrRNAとアニールした、請求項1に記載のガイド化合物。
- tracrRNAが、肺炎レンサ球菌、化膿レンサ球菌、髄膜炎菌またはサーモフィラス菌由来である、請求項18に記載のガイド化合物。
- tracrRNAが配列番号606である、請求項18に記載のガイド化合物。
- tracrRNAとアニールし、CRISPR関連遺伝子編集タンパク質と複合体を形成している、請求項1に記載のガイド化合物。
- CRISPR関連遺伝子編集タンパク質がCas9である、請求項21に記載のガイド化合物。
- 請求項18に記載の1以上のガイド化合物および薬学的に許容し得る担体を含む、医薬組成物。
- 薬学的に許容し得る担体が、ウイルスベクターまたは非ウイルスベクターを含む、請求項23に記載の組成物。
- 薬学的に許容し得る担体がリポソームを含む、請求項23に記載の組成物。
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562221407P | 2015-09-21 | 2015-09-21 | |
US62/221,407 | 2015-09-21 | ||
US201662330827P | 2016-05-02 | 2016-05-02 | |
US62/330,827 | 2016-05-02 | ||
PCT/US2016/052889 WO2017053431A2 (en) | 2015-09-21 | 2016-09-21 | Allele selective gene editing and uses thereof |
Publications (3)
Publication Number | Publication Date |
---|---|
JP2018527013A JP2018527013A (ja) | 2018-09-20 |
JP2018527013A5 true JP2018527013A5 (ja) | 2019-10-31 |
JP6799058B2 JP6799058B2 (ja) | 2020-12-09 |
Family
ID=58276126
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2018515082A Active JP6799058B2 (ja) | 2015-09-21 | 2016-09-21 | アレル選択的な遺伝子編集およびその使用 |
Country Status (7)
Country | Link |
---|---|
US (1) | US10369232B2 (ja) |
EP (1) | EP3353298B1 (ja) |
JP (1) | JP6799058B2 (ja) |
CN (1) | CN108350454B (ja) |
CA (1) | CA3002647A1 (ja) |
ES (1) | ES2959815T3 (ja) |
WO (1) | WO2017053431A2 (ja) |
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2016
- 2016-09-21 US US15/272,107 patent/US10369232B2/en active Active
- 2016-09-21 WO PCT/US2016/052889 patent/WO2017053431A2/en active Application Filing
- 2016-09-21 JP JP2018515082A patent/JP6799058B2/ja active Active
- 2016-09-21 CN CN201680067107.5A patent/CN108350454B/zh active Active
- 2016-09-21 CA CA3002647A patent/CA3002647A1/en active Pending
- 2016-09-21 EP EP16849508.3A patent/EP3353298B1/en active Active
- 2016-09-21 ES ES16849508T patent/ES2959815T3/es active Active
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