JP7596259B2 - プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 - Google Patents
プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 Download PDFInfo
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- JP7596259B2 JP7596259B2 JP2021513765A JP2021513765A JP7596259B2 JP 7596259 B2 JP7596259 B2 JP 7596259B2 JP 2021513765 A JP2021513765 A JP 2021513765A JP 2021513765 A JP2021513765 A JP 2021513765A JP 7596259 B2 JP7596259 B2 JP 7596259B2
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
- C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
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- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024095566A JP2024138268A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862670588P | 2018-05-11 | 2018-05-11 | |
| US62/670,588 | 2018-05-11 | ||
| US201862780838P | 2018-12-17 | 2018-12-17 | |
| US62/780,838 | 2018-12-17 | ||
| US201962817986P | 2019-03-13 | 2019-03-13 | |
| US62/817,986 | 2019-03-13 | ||
| PCT/US2019/031899 WO2019217944A1 (en) | 2018-05-11 | 2019-05-11 | Methods of editing single nucleotide polymorphism using programmable base editor systems |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024095566A Division JP2024138268A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021523739A JP2021523739A (ja) | 2021-09-09 |
| JPWO2019217944A5 JPWO2019217944A5 (enExample) | 2022-05-19 |
| JP7596259B2 true JP7596259B2 (ja) | 2024-12-09 |
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Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021513765A Active JP7596259B2 (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
| JP2021513764A Pending JP2021523738A (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
| JP2024095618A Pending JP2024147545A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
| JP2024095566A Pending JP2024138268A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021513764A Pending JP2021523738A (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
| JP2024095618A Pending JP2024147545A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
| JP2024095566A Pending JP2024138268A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US20210380955A1 (enExample) |
| EP (2) | EP3790963A4 (enExample) |
| JP (4) | JP7596259B2 (enExample) |
| KR (3) | KR102853088B1 (enExample) |
| CN (3) | CN119587720A (enExample) |
| AU (2) | AU2019266327B2 (enExample) |
| CA (2) | CA3100034A1 (enExample) |
| WO (2) | WO2019217943A1 (enExample) |
Families Citing this family (56)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9944912B2 (en) | 2015-03-03 | 2018-04-17 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| US11932856B2 (en) | 2017-03-03 | 2024-03-19 | The Regents Of The University Of California | RNA targeting of mutations via suppressor tRNAs and deaminases |
| EP3633034A4 (en) * | 2017-05-31 | 2021-03-24 | The University Of Tokyo | MODIFIED Cas9 PROTEIN AND USE THEREOF |
| CA3073448A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| WO2019111258A1 (en) * | 2017-12-07 | 2019-06-13 | Ramot At Tel-Aviv University Ltd. | Treatment for parkinsonian patients with mutations in the lrrk2 gene |
| WO2019178427A1 (en) | 2018-03-14 | 2019-09-19 | Arbor Biotechnologies, Inc. | Novel crispr dna targeting enzymes and systems |
| US12133884B2 (en) | 2018-05-11 | 2024-11-05 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US12157760B2 (en) * | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12454694B2 (en) | 2018-09-07 | 2025-10-28 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| AU2020221355A1 (en) * | 2019-02-13 | 2021-08-12 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
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