BR112022015969A2 - Composições e métodos para enxerto de células editadas de base - Google Patents
Composições e métodos para enxerto de células editadas de baseInfo
- Publication number
- BR112022015969A2 BR112022015969A2 BR112022015969A BR112022015969A BR112022015969A2 BR 112022015969 A2 BR112022015969 A2 BR 112022015969A2 BR 112022015969 A BR112022015969 A BR 112022015969A BR 112022015969 A BR112022015969 A BR 112022015969A BR 112022015969 A2 BR112022015969 A2 BR 112022015969A2
- Authority
- BR
- Brazil
- Prior art keywords
- methods
- compositions
- graftting
- base cells
- edited base
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/795—Porphyrin- or corrin-ring-containing peptides
- C07K14/805—Haemoglobins; Myoglobins
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Hematology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Developmental Biology & Embryology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
COMPOSIÇÕES E MÉTODOS PARA ENXERTO DE CÉLULAS EDITADAS DE BASE. A invenção fornece composições compreendendo novos editores de base de adenosina (por exemplo, ABE8) que aumentaram eficiência e os métodos de uso dessas variantes de adenosina desaminase para editar uma sequência alvo e os métodos de uso das mesmas para tratar distúrbios ou condições genéticas, por exemplo, doença falciforme, com enxerto.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202062976239P | 2020-02-13 | 2020-02-13 | |
PCT/US2021/017989 WO2021163587A1 (en) | 2020-02-13 | 2021-02-12 | Compositions and methods for engraftment of base edited cells |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112022015969A2 true BR112022015969A2 (pt) | 2022-10-25 |
Family
ID=77291886
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022015969A BR112022015969A2 (pt) | 2020-02-13 | 2021-02-12 | Composições e métodos para enxerto de células editadas de base |
Country Status (7)
Country | Link |
---|---|
US (1) | US20230242884A1 (pt) |
EP (1) | EP4103704A1 (pt) |
CN (1) | CN116113692A (pt) |
AU (1) | AU2021218811A1 (pt) |
BR (1) | BR112022015969A2 (pt) |
CA (1) | CA3170326A1 (pt) |
WO (1) | WO2021163587A1 (pt) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN116497067A (zh) | 2019-02-13 | 2023-07-28 | 比姆医疗股份有限公司 | 治疗血红素病变的组合物和方法 |
CN110982820A (zh) * | 2020-01-03 | 2020-04-10 | 云南中烟工业有限责任公司 | 一种烟草单倍体的基因编辑方法 |
WO2022232839A1 (en) * | 2021-04-30 | 2022-11-03 | The Board Of Trustees Of The Leland Stanford Junior University | Methods for improved production of primary cd34+ cells |
EP4337701A1 (en) | 2021-05-10 | 2024-03-20 | Mammoth Biosciences, Inc. | Effector proteins and methods of use |
WO2023056139A1 (en) * | 2021-10-01 | 2023-04-06 | The Regents Of The University Of California | Compositions and methods for treating a β-thalassemia disease |
WO2024073606A1 (en) * | 2022-09-28 | 2024-04-04 | Regeneron Pharmaceuticals, Inc. | Antibody resistant modified receptors to enhance cell-based therapies |
CN117965505A (zh) * | 2023-06-28 | 2024-05-03 | 微光基因(苏州)有限公司 | 工程化的腺苷脱氨酶及碱基编辑器 |
Family Cites Families (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2017306676B2 (en) * | 2016-08-03 | 2024-02-22 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
JP2020521446A (ja) * | 2017-05-25 | 2020-07-27 | ザ ジェネラル ホスピタル コーポレイション | 二部塩基エディター(bbe)構造およびii型−c−cas9ジンクフィンガー編集 |
EP3765614A1 (en) * | 2018-03-14 | 2021-01-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
WO2019217942A1 (en) * | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
KR20210041008A (ko) * | 2018-08-03 | 2021-04-14 | 빔 테라퓨틱스, 인크. | 핵산 표적 서열을 변형시키기 위한 다중-이펙터 핵염기 편집기 및 이를 이용하는 방법 |
-
2021
- 2021-02-12 EP EP21753486.6A patent/EP4103704A1/en active Pending
- 2021-02-12 WO PCT/US2021/017989 patent/WO2021163587A1/en unknown
- 2021-02-12 BR BR112022015969A patent/BR112022015969A2/pt unknown
- 2021-02-12 CN CN202180028355.XA patent/CN116113692A/zh active Pending
- 2021-02-12 CA CA3170326A patent/CA3170326A1/en active Pending
- 2021-02-12 AU AU2021218811A patent/AU2021218811A1/en active Pending
- 2021-02-12 US US17/799,159 patent/US20230242884A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
EP4103704A1 (en) | 2022-12-21 |
CA3170326A1 (en) | 2021-08-19 |
CN116113692A (zh) | 2023-05-12 |
WO2021163587A1 (en) | 2021-08-19 |
US20230242884A1 (en) | 2023-08-03 |
AU2021218811A1 (en) | 2022-09-01 |
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