CL2017003411A1 - Tratamientos basados en crispr / cas9 - Google Patents
Tratamientos basados en crispr / cas9Info
- Publication number
- CL2017003411A1 CL2017003411A1 CL2017003411A CL2017003411A CL2017003411A1 CL 2017003411 A1 CL2017003411 A1 CL 2017003411A1 CL 2017003411 A CL2017003411 A CL 2017003411A CL 2017003411 A CL2017003411 A CL 2017003411A CL 2017003411 A1 CL2017003411 A1 CL 2017003411A1
- Authority
- CL
- Chile
- Prior art keywords
- crispr
- cas9
- methods
- repair
- additionally
- Prior art date
Links
- 108091033409 CRISPR Proteins 0.000 title abstract 3
- 101150038500 cas9 gene Proteins 0.000 title 1
- 238000011282 treatment Methods 0.000 title 1
- 238000000034 method Methods 0.000 abstract 3
- 238000010354 CRISPR gene editing Methods 0.000 abstract 2
- 108020004414 DNA Proteins 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 108091092878 Microsatellite Proteins 0.000 abstract 1
- 101710163270 Nuclease Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 239000004744 fabric Substances 0.000 abstract 1
Classifications
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- 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 [CRISPR]
-
- 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)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Mycology (AREA)
- Ophthalmology & Optometry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Epidemiology (AREA)
- Rheumatology (AREA)
- Pulmonology (AREA)
- Pain & Pain Management (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562188013P | 2015-07-02 | 2015-07-02 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2017003411A1 true CL2017003411A1 (es) | 2018-08-17 |
Family
ID=57609222
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2017003411A CL2017003411A1 (es) | 2015-07-02 | 2017-12-27 | Tratamientos basados en crispr / cas9 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20200010854A1 (https=) |
| EP (1) | EP3317409A4 (https=) |
| JP (1) | JP2018520149A (https=) |
| KR (1) | KR20180041120A (https=) |
| CN (1) | CN108350446A (https=) |
| AU (1) | AU2016287836A1 (https=) |
| BR (1) | BR112017028201A2 (https=) |
| CA (1) | CA2989331A1 (https=) |
| CL (1) | CL2017003411A1 (https=) |
| EA (1) | EA201890203A1 (https=) |
| IL (1) | IL256279A (https=) |
| MX (1) | MX2017016921A (https=) |
| WO (1) | WO2017004616A1 (https=) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| DK3155101T3 (da) | 2014-06-16 | 2020-05-04 | Univ Johns Hopkins | Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 遺伝子編集のための進化したＣａｓ９蛋白質 |
| ES2905558T3 (es) * | 2015-11-13 | 2022-04-11 | Avellino Lab Usa Inc | Procedimientos para el tratamiento de las distrofias corneales |
| EP3445375A1 (en) * | 2016-04-22 | 2019-02-27 | Intellia Therapeutics, Inc. | Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four |
| KR102547316B1 (ko) | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JP2020510038A (ja) | 2017-03-09 | 2020-04-02 | プレジデント アンド フェローズ オブ ハーバード カレッジ | がんワクチン |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| WO2018165541A1 (en) * | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
| 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 |
| CN111065736A (zh) * | 2017-06-07 | 2020-04-24 | 国立大学法人东京大学 | 针对颗粒状角膜变性症的基因治疗药物 |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| DE102017131324A1 (de) | 2017-12-27 | 2019-06-27 | Beckhoff Automation Gmbh | Statormodul und Planarantriebssystem |
| CN108949823B (zh) * | 2017-12-29 | 2020-02-14 | 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） | 靶向敲除ATXN3基因中扩展突变型polyQ序列的方法 |
| US12480140B2 (en) | 2018-05-06 | 2025-11-25 | Emendobio Inc. | Differential knockout of an allele of a heterozygous ELANE gene |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| KR102126573B1 (ko) | 2018-10-18 | 2020-06-26 | 대한민국 | CRISPR-Cas9 시스템을 이용한 유색고약버섯의 리그닌 분해효소의 유전자 편집방법 및 이의 용도 |
| JP7216877B2 (ja) * | 2018-10-29 | 2023-02-02 | 中国▲農▼▲業▼大学 | 新規なＣＲＩＳＰＲ／Ｃａｓ１２ｆ酵素およびシステム |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| JP2022551970A (ja) * | 2019-10-16 | 2022-12-14 | ブラウン ユニバーシティ | 筋再生および筋成長 |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| CN111849991B (zh) * | 2020-08-05 | 2022-04-08 | 武汉纽福斯生物科技有限公司 | 一种寡核苷酸及其应用 |
| EP4430185A1 (en) * | 2021-11-09 | 2024-09-18 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy |
| WO2023092132A1 (en) * | 2021-11-22 | 2023-05-25 | Mammoth Biosciences, Inc. | Effector proteins and uses thereof |
| WO2024097900A1 (en) * | 2022-11-02 | 2024-05-10 | Emendobio Inc. | Compositions and methods for transcription factor 4 (tcf4) repeat expansion excision |
| CN116286823A (zh) * | 2023-04-04 | 2023-06-23 | 尧唐(上海)生物科技有限公司 | 靶向TGFBI基因的sgRNA、载体及其应用 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2931898T3 (en) * | 2012-12-12 | 2016-06-20 | Massachusetts Inst Technology | CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| AU2014361781B2 (en) * | 2013-12-12 | 2021-04-01 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing |
| MX2016007325A (es) * | 2013-12-12 | 2017-07-19 | Broad Inst Inc | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
| EP3445375A1 (en) * | 2016-04-22 | 2019-02-27 | Intellia Therapeutics, Inc. | Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four |
-
2016
- 2016-07-05 CA CA2989331A patent/CA2989331A1/en not_active Abandoned
- 2016-07-05 BR BR112017028201A patent/BR112017028201A2/pt not_active Application Discontinuation
- 2016-07-05 WO PCT/US2016/040962 patent/WO2017004616A1/en not_active Ceased
- 2016-07-05 MX MX2017016921A patent/MX2017016921A/es unknown
- 2016-07-05 EA EA201890203A patent/EA201890203A1/ru unknown
- 2016-07-05 EP EP16818953.8A patent/EP3317409A4/en not_active Withdrawn
- 2016-07-05 AU AU2016287836A patent/AU2016287836A1/en not_active Abandoned
- 2016-07-05 JP JP2017567629A patent/JP2018520149A/ja active Pending
- 2016-07-05 CN CN201680050474.4A patent/CN108350446A/zh active Pending
- 2016-07-05 US US15/741,444 patent/US20200010854A1/en not_active Abandoned
- 2016-07-05 KR KR1020187002971A patent/KR20180041120A/ko not_active Withdrawn
-
2017
- 2017-12-12 IL IL256279A patent/IL256279A/en unknown
- 2017-12-27 CL CL2017003411A patent/CL2017003411A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EA201890203A1 (ru) | 2018-07-31 |
| MX2017016921A (es) | 2018-04-10 |
| EP3317409A1 (en) | 2018-05-09 |
| CA2989331A1 (en) | 2017-01-05 |
| KR20180041120A (ko) | 2018-04-23 |
| CN108350446A (zh) | 2018-07-31 |
| AU2016287836A1 (en) | 2018-02-15 |
| BR112017028201A2 (pt) | 2018-08-28 |
| IL256279A (en) | 2018-02-28 |
| US20200010854A1 (en) | 2020-01-09 |
| EP3317409A4 (en) | 2019-02-20 |
| WO2017004616A1 (en) | 2017-01-05 |
| JP2018520149A (ja) | 2018-07-26 |
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