JPWO2019186275A5 - - Google Patents
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- JPWO2019186275A5 JPWO2019186275A5 JP2021501114A JP2021501114A JPWO2019186275A5 JP WO2019186275 A5 JPWO2019186275 A5 JP WO2019186275A5 JP 2021501114 A JP2021501114 A JP 2021501114A JP 2021501114 A JP2021501114 A JP 2021501114A JP WO2019186275 A5 JPWO2019186275 A5 JP WO2019186275A5
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- 108020004707 nucleic acids Proteins 0.000 claims 18
- 102000039446 nucleic acids Human genes 0.000 claims 18
- 150000007523 nucleic acids Chemical class 0.000 claims 18
- 210000004027 cell Anatomy 0.000 claims 13
- 230000000694 effects Effects 0.000 claims 7
- 108090000765 processed proteins & peptides Proteins 0.000 claims 6
- 206010028980 Neoplasm Diseases 0.000 claims 5
- 201000011510 cancer Diseases 0.000 claims 5
- 229920001184 polypeptide Polymers 0.000 claims 5
- 102000004196 processed proteins & peptides Human genes 0.000 claims 5
- 238000003776 cleavage reaction Methods 0.000 claims 4
- 230000007017 scission Effects 0.000 claims 4
- 108700004991 Cas12a Proteins 0.000 claims 3
- 108060002716 Exonuclease Proteins 0.000 claims 3
- 101710163270 Nuclease Proteins 0.000 claims 3
- 102000013165 exonuclease Human genes 0.000 claims 3
- 230000001939 inductive effect Effects 0.000 claims 3
- 108091033409 CRISPR Proteins 0.000 claims 2
- 208000031404 Chromosome Aberrations Diseases 0.000 claims 2
- 102100031780 Endonuclease Human genes 0.000 claims 2
- 108010042407 Endonucleases Proteins 0.000 claims 2
- 101001012157 Homo sapiens Receptor tyrosine-protein kinase erbB-2 Proteins 0.000 claims 2
- -1 LINK00536 Proteins 0.000 claims 2
- 102100030086 Receptor tyrosine-protein kinase erbB-2 Human genes 0.000 claims 2
- 230000030833 cell death Effects 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 230000005945 translocation Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- WEVYNIUIFUYDGI-UHFFFAOYSA-N 3-[6-[4-(trifluoromethoxy)anilino]-4-pyrimidinyl]benzamide Chemical compound NC(=O)C1=CC=CC(C=2N=CN=C(NC=3C=CC(OC(F)(F)F)=CC=3)C=2)=C1 WEVYNIUIFUYDGI-UHFFFAOYSA-N 0.000 claims 1
- 102100032291 A disintegrin and metalloproteinase with thrombospondin motifs 16 Human genes 0.000 claims 1
- 108091005675 ADAMTS16 Proteins 0.000 claims 1
- 206010006187 Breast cancer Diseases 0.000 claims 1
- 208000026310 Breast neoplasm Diseases 0.000 claims 1
- 102100035875 C-C chemokine receptor type 5 Human genes 0.000 claims 1
- 101710149870 C-C chemokine receptor type 5 Proteins 0.000 claims 1
- 206010008342 Cervix carcinoma Diseases 0.000 claims 1
- 206010009944 Colon cancer Diseases 0.000 claims 1
- 102000009512 Cyclin-Dependent Kinase Inhibitor p15 Human genes 0.000 claims 1
- 108010009356 Cyclin-Dependent Kinase Inhibitor p15 Proteins 0.000 claims 1
- 101700024220 DACH2 Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 102000004594 DNA Polymerase I Human genes 0.000 claims 1
- 108010017826 DNA Polymerase I Proteins 0.000 claims 1
- 102100025694 Dachshund homolog 2 Human genes 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 108010007577 Exodeoxyribonuclease I Proteins 0.000 claims 1
- 102100036702 Glucosamine-6-phosphate isomerase 2 Human genes 0.000 claims 1
- 102100029144 Histone-lysine N-methyltransferase PRDM9 Human genes 0.000 claims 1
- 101000896557 Homo sapiens Eukaryotic translation initiation factor 3 subunit B Proteins 0.000 claims 1
- 101001072480 Homo sapiens Glucosamine-6-phosphate isomerase 2 Proteins 0.000 claims 1
- 101001124887 Homo sapiens Histone-lysine N-methyltransferase PRDM9 Proteins 0.000 claims 1
- 101000988834 Homo sapiens Hypoxanthine-guanine phosphoribosyltransferase Proteins 0.000 claims 1
- 101001053444 Homo sapiens Iroquois-class homeodomain protein IRX-1 Proteins 0.000 claims 1
- 101000614442 Homo sapiens Keratin, type I cytoskeletal 16 Proteins 0.000 claims 1
- 101001139062 Homo sapiens Kinesin heavy chain isoform 5A Proteins 0.000 claims 1
- 101000741880 Homo sapiens POTE ankyrin domain family member B Proteins 0.000 claims 1
- 101000923322 Homo sapiens Phospholipid-transporting ATPase IH Proteins 0.000 claims 1
- 101000974720 Homo sapiens Potassium voltage-gated channel subfamily E member 2 Proteins 0.000 claims 1
- 101000984753 Homo sapiens Serine/threonine-protein kinase B-raf Proteins 0.000 claims 1
- 101000709256 Homo sapiens Signal-regulatory protein beta-1 Proteins 0.000 claims 1
- 101000709188 Homo sapiens Signal-regulatory protein beta-1 isoform 3 Proteins 0.000 claims 1
- 101000688924 Homo sapiens Small integral membrane protein 11 Proteins 0.000 claims 1
- 101000772560 Homo sapiens Zinc finger transcription factor Trps1 Proteins 0.000 claims 1
- 102100029098 Hypoxanthine-guanine phosphoribosyltransferase Human genes 0.000 claims 1
- 102100024435 Iroquois-class homeodomain protein IRX-1 Human genes 0.000 claims 1
- 102100040441 Keratin, type I cytoskeletal 16 Human genes 0.000 claims 1
- 102100020685 Kinesin heavy chain isoform 5A Human genes 0.000 claims 1
- 206010058467 Lung neoplasm malignant Diseases 0.000 claims 1
- 102100025354 Macrophage mannose receptor 1 Human genes 0.000 claims 1
- 108010031099 Mannose Receptor Proteins 0.000 claims 1
- 206010033128 Ovarian cancer Diseases 0.000 claims 1
- 206010061535 Ovarian neoplasm Diseases 0.000 claims 1
- 102100038746 POTE ankyrin domain family member B Human genes 0.000 claims 1
- 206010061902 Pancreatic neoplasm Diseases 0.000 claims 1
- 102100032688 Phospholipid-transporting ATPase IH Human genes 0.000 claims 1
- 102100022752 Potassium voltage-gated channel subfamily E member 2 Human genes 0.000 claims 1
- 108091006596 SLC15A5 Proteins 0.000 claims 1
- 102100027103 Serine/threonine-protein kinase B-raf Human genes 0.000 claims 1
- 102100032770 Signal-regulatory protein beta-1 isoform 3 Human genes 0.000 claims 1
- 102100024468 Small integral membrane protein 11 Human genes 0.000 claims 1
- 102100021492 Solute carrier family 15 member 5 Human genes 0.000 claims 1
- 208000006105 Uterine Cervical Neoplasms Diseases 0.000 claims 1
- 102100030619 Zinc finger transcription factor Trps1 Human genes 0.000 claims 1
- 201000010881 cervical cancer Diseases 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 210000000349 chromosome Anatomy 0.000 claims 1
- 208000029742 colonic neoplasm Diseases 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 201000005202 lung cancer Diseases 0.000 claims 1
- 208000020816 lung neoplasm Diseases 0.000 claims 1
- 208000015486 malignant pancreatic neoplasm Diseases 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 201000002528 pancreatic cancer Diseases 0.000 claims 1
- 208000008443 pancreatic carcinoma Diseases 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
Claims (31)
- 細胞中で細胞死を選択的に誘導する際に使用される薬剤の製造における、配列特異的なエンドヌクレアーゼ活性を含む第１のドメインと、エキソヌクレアーゼ活性を含む第２のドメインとを含むキメラポリペプチド、および細胞中の標的核酸と相補的な配列を含むガイド核酸の使用であって、
誘導は、
ａ）配列特異的なエンドヌクレアーゼ活性を含む第１のドメインと、エキソヌクレアーゼ活性を含む第２のドメインとを含むキメラポリペプチド、および細胞中の標的核酸と相補的な配列を含むガイド核酸を、被験体に投与すること；および、
ｂ）標的核酸を切断することであって、それによって、細胞死を誘導する、こと、を含む、使用。 - 標的核酸は疾患に関連付けられる、請求項１に記載の使用。
- 標的核酸は被験体の癌細胞中にある、請求項１に記載の使用。
- 標的核酸は被験体の正常細胞にはない、請求項１に記載の使用。
- 標的核酸は癌に関連する配列を含む、請求項１に記載の使用。
- 標的核酸はＲＮＡを含む、請求項１に記載の使用。
- 標的核酸はＤＮＡを含む、請求項１に記載の使用。
- 標的核酸は染色体異常を含む領域内にある、請求項１に記載の使用。
- 染色体異常は、転座、欠失、重複、逆位、挿入、環、コピー数多型、インデル、および同腕染色体からなる群から選択される、請求項８に記載の使用。
- 細胞は複数の細胞にある、請求項１に記載の使用。
- 複数の細胞は正常細胞を含む、請求項１０に記載の使用。
- 投与後、正常細胞は生きている、請求項１１に記載の使用。
- 投与後、正常細胞は増殖する、請求項１１に記載の使用。
- 前記切断することは、細胞中の１つ以上の切断部位での切断を含む、請求項１に記載の使用。
- 標的核酸は疾患に関連付けられる、請求項１に記載の使用。
- 癌は、肺癌、膵臓癌、乳癌、卵巣癌、大腸癌、または子宮頚癌を含む、請求項５に記載の使用。
- 標的核酸は、癌、転座、または癌の進行に関連する配列に特異的な一塩基多型を含む、請求項１に記載の使用。
- 標的核酸は、ＢＲＣＡ－１、ＢＲＡＦ、ＢＣＲ－ＡＢＬ、ＨＥＲ２、ＫＩＦ５Ａ、ＩＲＸ１、ＡＤＡＭＴＳ１６、ＧＮＰＤＡ２、ＫＣＮＥ２、ＳＬＣ１５Ａ５、ＳＭＩＭ１１、ＤＡＣＨ２、ＨＥＲＣ２Ｐ２、ＣＤ６８ＳＨＢＧ、ＥＲＢＢ２、ＫＲＴ１６、ＬＩＮＣ００５３６、ＴＲＰＳ１、ＣＤＫ８、ＴＲＡＰＰＣ９、ＨＥＲＣ２Ｐ２、ＳＩＲＰＢ１、ＭＲＣ１、ＡＴＰ１１Ａ、ＰＯＴＥＢ、ＨＥＲＣ２Ｐ２、ＰＲＤＭ９、ＣＤＫＮ２Ｂ、ＨＰＶ、ＬＩＮＥ２ （ＭＴ２）、ＣＣＲ５、またはＨＰＲＴ１からなる群から選択された遺伝子の一部を含む、請求項１に記載の使用。
- キメラポリペプチドは誘導可能な非特異的ヌクレアーゼ活性をさらに含む、請求項１に記載の使用。
- 誘導可能な非特異的ヌクレアーゼ活性は、キメラポリペプチドによる標的核酸の部位特異的な切断によって活性化される、請求項１９に記載の使用。
- 第１のドメインは、Ｃａｓ１２ａ（またはＣｐｆ１）ドメイン、Ｃａｓ１２ａドメイン、Ｃａｓ１２ｂドメイン、Ｃａｓ１２ｃドメイン、Ｃａｓ１２ｄドメイン、Ｃａｓ１２ｅドメイン、Ｃａｓ１２ｆドメイン、Ｃａｓ１２ｇドメイン、Ｃａｓ１２ｈドメイン、Ｃａｓ１２ｉドメイン、Ｃａｓ１３ａドメイン、Ｃａｓ１３ｂドメイン、Ｃａｓ１４ドメイン、またはＣａｓ９ドメインを含む、請求項１に記載の使用。
- 第１のドメインはＣａｓ１２ａ（またはＣｐｆ１）ドメインを含む、請求項１に記載の使用。
- 第１のドメインはＣａｓ１３ａまたはＣａｓ１３ｂのドメインを含む、請求項１に記載の使用。
- 第１のドメインはＣａｓ９ドメインを含む、請求項１に記載の使用。
- ガイド核酸は、表６と段落［０２６８］、あるいは表７と段落［０２７０］に表記された配列からなる群から選択された配列を含む、請求項１に記載の使用。
- 第２のドメインは、ＲｅｃＥドメイン、ＲｅｃＪドメイン、Ｔ５ドメイン、ラムダエキソヌクレアーゼドメイン、ＲｅｃＢＣＤドメイン、ＤＮＡポリメラーゼＩ小断片ドメイン、ＥｘｏＩドメイン、ＥｘｏＩＩドメイン、ＥｘｏＶＩＩドメイン、Ｅｘｏ Ｔドメイン、Ｔｒｅｘ１ドメイン、またはＴｒｅｘ２ドメインを含む、請求項１に記載の使用。
- キメラポリペプチドは３’ＯＨオーバーハングを生成する、請求項１に記載の使用。
- キメラポリペプチドは陥凹３’ＯＨを露出させる、請求項１に記載の使用。
- 第２のドメインは切断された末端切除活性を有する酵素を含む、請求項１に記載の使用。
- 第２のドメインはマングビーンヌクレアーゼを含む、請求項１に記載の使用。
- 標的核酸は被験体の正常細胞にはない、請求項１１に記載の使用。
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862652150P | 2018-04-03 | 2018-04-03 | |
| US62/652,150 | 2018-04-03 | ||
| US201862724199P | 2018-08-29 | 2018-08-29 | |
| US62/724,199 | 2018-08-29 | ||
| PCT/IB2019/000346 WO2019186275A1 (en) | 2018-03-27 | 2019-03-27 | Sequence-specific in vivo cell targeting |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022518643A JP2022518643A (ja) | 2022-03-16 |
| JPWO2019186275A5 true JPWO2019186275A5 (ja) | 2022-04-04 |
| JP7422128B2 JP7422128B2 (ja) | 2024-01-25 |
Family
ID=80668828
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021501114A Active JP7422128B2 (ja) | 2018-04-03 | 2019-03-27 | 配列特異的なインビボ細胞標的化 |
Country Status (1)
| Country | Link |
|---|---|
| JP (1) | JP7422128B2 (ja) |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007049095A1 (en) * | 2005-10-25 | 2007-05-03 | Cellectis | Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof |
| WO2008093152A1 (en) * | 2007-02-01 | 2008-08-07 | Cellectis | Obligate heterodimer meganucleases and uses thereof |
| GB201122458D0 (en) * | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| EP3786294A1 (en) * | 2015-09-24 | 2021-03-03 | Editas Medicine, Inc. | Use of exonucleases to improve crispr/cas-mediated genome editing |
| AU2017308889B2 (en) * | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
-
2019
- 2019-03-27 JP JP2021501114A patent/JP7422128B2/ja active Active
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