JP2019531068A5 - - Google Patents
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- JP2019531068A5 JP2019531068A5 JP2019512750A JP2019512750A JP2019531068A5 JP 2019531068 A5 JP2019531068 A5 JP 2019531068A5 JP 2019512750 A JP2019512750 A JP 2019512750A JP 2019512750 A JP2019512750 A JP 2019512750A JP 2019531068 A5 JP2019531068 A5 JP 2019531068A5
- Authority
- JP
- Japan
- Prior art keywords
- domain
- fusion molecule
- gene
- hepatocyte
- dna binding
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
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- 210000003494 hepatocyte Anatomy 0.000 claims description 16
- 108090000623 proteins and genes Proteins 0.000 claims description 16
- 230000004927 fusion Effects 0.000 claims description 12
- 230000004568 DNA-binding Effects 0.000 claims description 10
- 108091033319 polynucleotide Proteins 0.000 claims description 6
- 102000040430 polynucleotide Human genes 0.000 claims description 6
- 239000002157 polynucleotide Substances 0.000 claims description 6
- 102100022712 Alpha-1-antitrypsin Human genes 0.000 claims description 5
- 102100034869 Plasma kallikrein Human genes 0.000 claims description 4
- 102100038955 Proprotein convertase subtilisin/kexin type 9 Human genes 0.000 claims description 4
- 108091023040 Transcription factor Proteins 0.000 claims description 4
- 102000040945 Transcription factor Human genes 0.000 claims description 4
- 238000003776 cleavage reaction Methods 0.000 claims description 4
- 230000035772 mutation Effects 0.000 claims description 4
- 239000008194 pharmaceutical composition Substances 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- 230000007017 scission Effects 0.000 claims description 4
- 230000002103 transcriptional effect Effects 0.000 claims description 4
- 101000823116 Homo sapiens Alpha-1-antitrypsin Proteins 0.000 claims description 3
- 108020005004 Guide RNA Proteins 0.000 claims description 2
- 101150105486 HAO1 gene Proteins 0.000 claims description 2
- 229940121710 HMGCoA reductase inhibitor Drugs 0.000 claims description 2
- 206010019860 Hereditary angioedema Diseases 0.000 claims description 2
- 101001031589 Homo sapiens 2-Hydroxyacid oxidase 1 Proteins 0.000 claims description 2
- 101001091365 Homo sapiens Plasma kallikrein Proteins 0.000 claims description 2
- 101001098868 Homo sapiens Proprotein convertase subtilisin/kexin type 9 Proteins 0.000 claims description 2
- 208000035150 Hypercholesterolemia Diseases 0.000 claims description 2
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 claims description 2
- 208000008852 Hyperoxaluria Diseases 0.000 claims description 2
- 101150038962 KLKB1 gene Proteins 0.000 claims description 2
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 101150094724 PCSK9 gene Proteins 0.000 claims description 2
- 101150086419 Serpina11 gene Proteins 0.000 claims description 2
- -1 TTR Proteins 0.000 claims description 2
- 101150091380 TTR gene Proteins 0.000 claims description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims description 2
- 101710185494 Zinc finger protein Proteins 0.000 claims description 2
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 2
- 206010002022 amyloidosis Diseases 0.000 claims description 2
- 230000007812 deficiency Effects 0.000 claims description 2
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims description 2
- 230000001404 mediated effect Effects 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 230000003612 virological effect Effects 0.000 claims description 2
- 210000005229 liver cell Anatomy 0.000 claims 2
- HYIMSNHJOBLJNT-UHFFFAOYSA-N nifedipine Chemical compound COC(=O)C1=C(C)NC(C)=C(C(=O)OC)C1C1=CC=CC=C1[N+]([O-])=O HYIMSNHJOBLJNT-UHFFFAOYSA-N 0.000 claims 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023057789A JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662384428P | 2016-09-07 | 2016-09-07 | |
| US62/384,428 | 2016-09-07 | ||
| PCT/US2017/050446 WO2018049009A2 (en) | 2016-09-07 | 2017-09-07 | Modulation of liver genes |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023057789A Division JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019531068A JP2019531068A (ja) | 2019-10-31 |
| JP2019531068A5 true JP2019531068A5 (enExample) | 2020-10-15 |
| JP7256739B2 JP7256739B2 (ja) | 2023-04-12 |
Family
ID=61281860
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019512750A Active JP7256739B2 (ja) | 2016-09-07 | 2017-09-07 | 肝臓遺伝子のモジュレーション |
| JP2023057789A Withdrawn JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023057789A Withdrawn JP2023082133A (ja) | 2016-09-07 | 2023-03-31 | 肝臓遺伝子のモジュレーション |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US10960085B2 (enExample) |
| EP (1) | EP3509645A4 (enExample) |
| JP (2) | JP7256739B2 (enExample) |
| AU (1) | AU2017324462B2 (enExample) |
| CA (1) | CA3035534A1 (enExample) |
| WO (1) | WO2018049009A2 (enExample) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
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| MX2019006426A (es) | 2016-12-01 | 2019-08-14 | Sangamo Therapeutics Inc | Reguladores de tau, y composiciones y metodos para su administracion. |
| US11820728B2 (en) | 2017-04-28 | 2023-11-21 | Acuitas Therapeutics, Inc. | Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| EP3585162B1 (en) | 2017-09-29 | 2023-08-30 | Regeneron Pharmaceuticals, Inc. | Rodents comprising a humanized ttr locus and methods of use |
| WO2020010186A1 (en) * | 2018-07-06 | 2020-01-09 | Derek Klarin | Pcsk9 variants |
| CA3113190A1 (en) * | 2018-07-31 | 2020-02-06 | Intellia Therapeutics, Inc. | Compositions and methods for hydroxyacid oxidase 1 (hao1) gene editing for treating primary hyperoxaluria type 1 (ph1) |
| EP3852911B1 (en) | 2018-09-21 | 2025-01-22 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| CN113226333A (zh) | 2018-10-02 | 2021-08-06 | 桑格摩生物治疗股份有限公司 | 用于调控Tau蛋白的方法和组合物 |
| EP3861130A4 (en) * | 2018-10-02 | 2022-08-03 | Sangamo Therapeutics, Inc. | GENETIC MODULATORS |
| BR112021007301A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para expressar fator ix |
| EP3898661A1 (en) * | 2018-12-21 | 2021-10-27 | Precision BioSciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| US11453639B2 (en) | 2019-01-11 | 2022-09-27 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| SG11202108568YA (en) * | 2019-02-25 | 2021-09-29 | Univ Massachusetts | Dna-binding domain transactivators and uses thereof |
| WO2020247452A1 (en) | 2019-06-04 | 2020-12-10 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use |
| WO2021158883A1 (en) * | 2020-02-07 | 2021-08-12 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized klkb1 locus and methods of use |
| MX2023000614A (es) | 2020-07-16 | 2023-02-13 | Acuitas Therapeutics Inc | Lipidos cationicos para usarse en nanoparticulas lipidicas. |
| TW202221119A (zh) * | 2020-07-24 | 2022-06-01 | 麻州大學 | Dna結合域轉活化子及其用途 |
| EP4244342A1 (en) | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| WO2022256655A2 (en) | 2021-06-04 | 2022-12-08 | Arbor Biotechnologies, Inc. | Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof |
| CA3231594A1 (en) * | 2021-09-08 | 2023-03-16 | Flagship Pioneering Innovations Vi, Llc | Serpina-modulating compositions and methods |
| IL313486A (en) | 2021-12-16 | 2024-08-01 | Acuitas Therapeutics Inc | Lipids for use in lipid nanoparticle formulations |
| US20250360224A1 (en) * | 2022-03-11 | 2025-11-27 | Epicrispr Biotechnologies, Inc. | Systems and methods for genetic modulation to treat liver disease |
| AU2023283464A1 (en) | 2022-06-07 | 2024-12-05 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| TW202444906A (zh) | 2023-03-29 | 2024-11-16 | 美商斯奎柏治療公司 | 用於靶向pcsk9之組合物及方法 |
| WO2024229020A2 (en) * | 2023-05-01 | 2024-11-07 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of pcsk9 expression |
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| US10724020B2 (en) | 2016-02-02 | 2020-07-28 | Sangamo Therapeutics, Inc. | Compositions for linking DNA-binding domains and cleavage domains |
| US10975393B2 (en) | 2016-08-24 | 2021-04-13 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
-
2017
- 2017-09-07 US US15/697,917 patent/US10960085B2/en not_active Expired - Fee Related
- 2017-09-07 EP EP17849517.2A patent/EP3509645A4/en not_active Withdrawn
- 2017-09-07 WO PCT/US2017/050446 patent/WO2018049009A2/en not_active Ceased
- 2017-09-07 AU AU2017324462A patent/AU2017324462B2/en not_active Expired - Fee Related
- 2017-09-07 CA CA3035534A patent/CA3035534A1/en active Pending
- 2017-09-07 JP JP2019512750A patent/JP7256739B2/ja active Active
-
2020
- 2020-11-12 US US17/096,502 patent/US20210060179A1/en not_active Abandoned
-
2023
- 2023-03-31 JP JP2023057789A patent/JP2023082133A/ja not_active Withdrawn
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