DK3289076T3 - Præcis sletning af kromosomale sekvenser in vivo - Google Patents
Præcis sletning af kromosomale sekvenser in vivo Download PDFInfo
- Publication number
- DK3289076T3 DK3289076T3 DK16789905.3T DK16789905T DK3289076T3 DK 3289076 T3 DK3289076 T3 DK 3289076T3 DK 16789905 T DK16789905 T DK 16789905T DK 3289076 T3 DK3289076 T3 DK 3289076T3
- Authority
- DK
- Denmark
- Prior art keywords
- vivo
- chromosomal sequences
- precise deletion
- deletion
- precise
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- 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
-
- 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
- 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
- C12N15/111—General methods applicable to biologically active non-coding 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)
-
- 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
-
- 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]
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Crystallography & Structural Chemistry (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562155838P | 2015-05-01 | 2015-05-01 | |
| PCT/US2016/030460 WO2016179112A1 (en) | 2015-05-01 | 2016-05-02 | Precise deletion of chromoscomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3289076T3 true DK3289076T3 (da) | 2022-01-17 |
Family
ID=57218596
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK16789905.3T DK3289076T3 (da) | 2015-05-01 | 2016-05-02 | Præcis sletning af kromosomale sekvenser in vivo |
Country Status (5)
| Country | Link |
|---|---|
| US (3) | US20180344817A1 (da) |
| EP (2) | EP4015633A1 (da) |
| DK (1) | DK3289076T3 (da) |
| ES (1) | ES2905181T3 (da) |
| WO (1) | WO2016179112A1 (da) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2012333134B2 (en) | 2011-07-22 | 2017-05-25 | John Paul Guilinger | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| MX2017015369A (es) | 2015-05-29 | 2018-07-06 | Regeneron Pharma | Animales no humanos que tienen una alteracion en un locus de c9orf72. |
| WO2017062605A1 (en) * | 2015-10-06 | 2017-04-13 | The Children's Hospital Of Philadelphia | Compositions and methods for treating fragile x syndrome and related syndromes |
| EP3365357B1 (en) | 2015-10-23 | 2024-02-14 | President and Fellows of Harvard College | Evolved cas9 proteins for gene editing |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| CN109476716A (zh) | 2016-03-23 | 2019-03-15 | 加利福尼亚大学董事会 | 治疗线粒体障碍的方法 |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| ES2989940T3 (es) * | 2016-06-29 | 2024-11-28 | Crispr Therapeutics Ag | Materiales y métodos para el tratamiento de la ataxia de Friedreich y otros trastornos relacionados |
| EP3494215A1 (en) | 2016-08-03 | 2019-06-12 | President and Fellows of Harvard College | Adenosine nucleobase editors and uses thereof |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018064600A1 (en) | 2016-09-30 | 2018-04-05 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
| US11306324B2 (en) | 2016-10-14 | 2022-04-19 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| EP3532615A1 (en) * | 2016-10-28 | 2019-09-04 | Genethon | Compositions and methods for the treatment of myotonic dystrophy |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| MX2020005860A (es) | 2017-12-06 | 2020-09-09 | Avidity Biosciences Inc | Composiciones y metodos de tratamiento de atrofia muscular y distrofia miotonica. |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| US20220033823A1 (en) * | 2018-08-22 | 2022-02-03 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene |
| EP3732291A1 (en) | 2018-12-20 | 2020-11-04 | Regeneron Pharmaceuticals, Inc. | Nuclease-mediated repeat expansion |
| DE112020001342T5 (de) | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
| WO2020219870A1 (en) * | 2019-04-26 | 2020-10-29 | The Children's Hospital Of Philadelphia | Crispr/cas9 gene editing of atxn2 for the treatment of spinocerebellar ataxia type 2 |
| AU2020268394A1 (en) | 2019-05-07 | 2022-01-06 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| WO2021188349A2 (en) * | 2020-03-16 | 2021-09-23 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| KR20220156867A (ko) | 2020-03-19 | 2022-11-28 | 어비디티 바이오사이언시스 인크. | 안면견갑상완 근이영양증의 치료용 조성물 및 방법 |
| JP2023527638A (ja) | 2020-03-27 | 2023-06-30 | アビディティー バイオサイエンシーズ，インク． | 顔面肩甲上腕型筋ジストロフィーを処置するための組成物および方法 |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| MX2023013352A (es) | 2021-05-10 | 2024-01-31 | Entrada Therapeutics Inc | Composiciones y métodos para terapéuticos intracelulares. |
| PE20242357A1 (es) | 2021-06-23 | 2024-12-16 | Entrada Therapeutics Inc | Compuestos antisentido y metodos para dirigirse a repeticiones cug |
| IL312243A (en) * | 2021-10-19 | 2024-06-01 | Prec Biosciences Inc | Gene editing methods to treat alpha-1 antitrypsin (AAT) deficiency |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US6015832A (en) | 1997-12-31 | 2000-01-18 | The Regents Of The University Of Michigan | Methods of inactivating bacteria including bacterial spores |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| AU2004208031B2 (en) | 2003-01-28 | 2009-10-08 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof. |
| EP2650366B1 (en) | 2005-10-18 | 2017-03-22 | Precision Biosciences | Rationally designed meganucleases with altered sequence specificity and DNA-binding affinity |
| WO2008043149A1 (en) * | 2006-10-12 | 2008-04-17 | Simons Haplomics Limited | Methods for obtaining information from genetic material |
| ES2732735T3 (es) | 2007-10-31 | 2019-11-25 | Prec Biosciences Inc | Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas |
| JP2011527906A (ja) | 2008-07-14 | 2011-11-10 | プレシジョン バイオサイエンシズ，インク． | Ｉ−ＣｒｅＩ由来メガヌクレアーゼの認識配列およびその使用 |
| WO2012138939A1 (en) * | 2011-04-05 | 2012-10-11 | Philippe Duchateau | New tale-protein scaffolds and uses thereof |
| ES2713529T3 (es) | 2011-06-01 | 2019-05-22 | Prec Biosciences Inc | Procedimientos y productos para la producción de líneas celulares de mamífero modificadas con transgenes amplificados |
| JP5798504B2 (ja) * | 2011-11-18 | 2015-10-21 | 王子ホールディングス株式会社 | 微細繊維状セルロースの製造方法、不織布の製造方法、微細繊維状セルロース、微細繊維状セルロース含有スラリー、不織布、および複合体 |
-
2016
- 2016-05-02 DK DK16789905.3T patent/DK3289076T3/da active
- 2016-05-02 WO PCT/US2016/030460 patent/WO2016179112A1/en active Application Filing
- 2016-05-02 EP EP21208516.1A patent/EP4015633A1/en active Pending
- 2016-05-02 EP EP16789905.3A patent/EP3289076B1/en active Active
- 2016-05-02 US US15/571,216 patent/US20180344817A1/en not_active Abandoned
- 2016-05-02 ES ES16789905T patent/ES2905181T3/es active Active
-
2021
- 2021-01-15 US US17/151,075 patent/US20210228693A1/en not_active Abandoned
-
2022
- 2022-06-17 US US17/843,496 patent/US20230201317A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP3289076A1 (en) | 2018-03-07 |
| US20230201317A1 (en) | 2023-06-29 |
| US20180344817A1 (en) | 2018-12-06 |
| ES2905181T3 (es) | 2022-04-07 |
| EP3289076A4 (en) | 2018-11-07 |
| WO2016179112A1 (en) | 2016-11-10 |
| US20210228693A1 (en) | 2021-07-29 |
| EP4015633A1 (en) | 2022-06-22 |
| EP3289076B1 (en) | 2021-11-17 |
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