JP2016537341A - ハンチントン病を処置するための方法および組成物 - Google Patents
ハンチントン病を処置するための方法および組成物 Download PDFInfo
- Publication number
- JP2016537341A JP2016537341A JP2016528904A JP2016528904A JP2016537341A JP 2016537341 A JP2016537341 A JP 2016537341A JP 2016528904 A JP2016528904 A JP 2016528904A JP 2016528904 A JP2016528904 A JP 2016528904A JP 2016537341 A JP2016537341 A JP 2016537341A
- Authority
- JP
- Japan
- Prior art keywords
- zfp
- htt
- cag
- gene
- domain
- 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.)
- Pending
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
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6811—Selection methods for production or design of target specific oligonucleotides or binding molecules
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/10—Peptides having 12 to 20 amino acids
-
- 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
- A61K38/164—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
-
- 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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
-
- 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
-
- 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
-
- 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
- C12N2320/34—Allele or polymorphism specific uses
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Virology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Analytical Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361902704P | 2013-11-11 | 2013-11-11 | |
| US61/902,704 | 2013-11-11 | ||
| PCT/US2014/064987 WO2015070212A1 (en) | 2013-11-11 | 2014-11-11 | Methods and compositions for treating huntington's disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019067000A Division JP2019116502A (ja) | 2013-11-11 | 2019-03-29 | ハンチントン病を処置するための方法および組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016537341A true JP2016537341A (ja) | 2016-12-01 |
| JP2016537341A5 JP2016537341A5 (OSRAM) | 2017-10-12 |
Family
ID=53042231
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016528904A Pending JP2016537341A (ja) | 2013-11-11 | 2014-11-11 | ハンチントン病を処置するための方法および組成物 |
| JP2019067000A Pending JP2019116502A (ja) | 2013-11-11 | 2019-03-29 | ハンチントン病を処置するための方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019067000A Pending JP2019116502A (ja) | 2013-11-11 | 2019-03-29 | ハンチントン病を処置するための方法および組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US10369201B2 (OSRAM) |
| EP (1) | EP3068905A4 (OSRAM) |
| JP (2) | JP2016537341A (OSRAM) |
| KR (1) | KR102431079B1 (OSRAM) |
| CN (1) | CN105934524A (OSRAM) |
| AU (1) | AU2014346424B2 (OSRAM) |
| BR (1) | BR112016010175A2 (OSRAM) |
| CA (1) | CA2929179A1 (OSRAM) |
| MX (1) | MX391904B (OSRAM) |
| NZ (1) | NZ719477A (OSRAM) |
| RU (1) | RU2693891C1 (OSRAM) |
| WO (1) | WO2015070212A1 (OSRAM) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200077529A (ko) * | 2017-10-24 | 2020-06-30 | 상가모 테라퓨틱스, 인코포레이티드 | 희귀 질환의 치료를 위한 방법 및 조성물 |
Families Citing this family (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| BR112014001244A2 (pt) | 2011-07-19 | 2017-02-21 | Wave Life Sciences Pte Ltd | métodos para a síntese de ácidos nucléicos funcionalizados |
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| ES2862073T3 (es) | 2012-07-13 | 2021-10-06 | Wave Life Sciences Ltd | Grupo auxiliar asimétrico |
| CN112007045A (zh) | 2012-07-13 | 2020-12-01 | 波涛生命科学有限公司 | 手性控制 |
| BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
| 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 |
| 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 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| CA2930015A1 (en) | 2013-11-07 | 2015-05-14 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| US10144933B2 (en) | 2014-01-15 | 2018-12-04 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| WO2016077689A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| US20160362667A1 (en) * | 2015-06-10 | 2016-12-15 | Caribou Biosciences, Inc. | CRISPR-Cas Compositions and Methods |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics Inc | composições e métodos de tratamento da doença de huntington |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | 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 |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| JP7588390B2 (ja) | 2016-10-14 | 2024-11-22 | 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 | 核酸塩基エディターのaav送達 |
| US12180499B2 (en) * | 2016-10-31 | 2024-12-31 | University Of Florida Research Foundation, Inc. | Compositions and methods for impeding transcription of expanded microsatellite repeats |
| CN106636388A (zh) * | 2016-12-15 | 2017-05-10 | 湖南中能荆卫生物科技有限公司 | 用于亨廷顿病诊断的基因及其应用以及由其编码的蛋白和蛋白的应用 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| 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 |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | 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 | ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| WO2019060726A1 (en) * | 2017-09-22 | 2019-03-28 | Genzyme Corporation | Variant rnai |
| 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 |
| US11421007B2 (en) * | 2018-04-18 | 2022-08-23 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (Htt) |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| JP2021532736A (ja) * | 2018-06-06 | 2021-12-02 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | 神経幹細胞組成物および神経変性障害を処置するための方法 |
| CN109112162B (zh) * | 2018-08-30 | 2021-10-15 | 西南大学 | 利用CRISPR/Cas9技术构建的亨廷顿病原位敲入小鼠模型及构建方法 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| CN113301909B (zh) * | 2019-01-15 | 2025-01-28 | 桑格摩生物治疗股份有限公司 | Htt阻遏物及其应用 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| EP3942042A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| MX2021012836A (es) | 2019-04-23 | 2021-12-10 | Sangamo Therapeutics Inc | Moduladores de la expresion del gen del marco de lectura abierto 72 del cromosoma 9 y usos de los mismos. |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CA3155921A1 (en) * | 2019-11-01 | 2021-05-06 | Alnylam Pharmaceuticals, Inc. | HUNTINGTINE (HTT) TARGETING RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| TW202132565A (zh) | 2019-11-01 | 2021-09-01 | 美商聖加莫治療股份有限公司 | Gin重組酶變異體 |
| CN110747208B (zh) * | 2019-11-28 | 2022-04-22 | 海南大学 | 一种木薯硝酸还原酶基因及其过量表达载体的构建和抗病应用 |
| AU2020407119A1 (en) * | 2019-12-20 | 2022-07-14 | AskBio Inc. | Methods for treating Huntington's disease |
| 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 |
| CN115786395A (zh) * | 2022-08-19 | 2023-03-14 | 南京启真基因工程有限公司 | 构建htt基因突变的亨廷顿舞蹈症模型猪核移植供体细胞的基因编辑系统及其应用 |
| CN120752255A (zh) * | 2023-01-05 | 2025-10-03 | 恩沐辑医药有限责任公司 | 神经元启动子及其用途 |
| AU2024206221A1 (en) * | 2023-01-05 | 2025-07-03 | Emugen Therapeutics Llc | Retro-aav and use in treating neurodegenerative diseases |
| WO2025004001A1 (en) | 2023-06-30 | 2025-01-02 | Takeda Pharmaceutical Company Limited | Htt repressors and uses thereof |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010530239A (ja) * | 2007-06-18 | 2010-09-09 | コミッサリア ア レネルジ アトミック | 変異および内因性野生型ハンチンチン遺伝子のsiRNAによる可逆的サイレンシングおよびハンチントン病の治療のためのその応用 |
| WO2013130824A1 (en) * | 2012-02-29 | 2013-09-06 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
Family Cites Families (102)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
| US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
| US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
| US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
| US5422251A (en) | 1986-11-26 | 1995-06-06 | Princeton University | Triple-stranded nucleic acids |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| AU7979491A (en) | 1990-05-03 | 1991-11-27 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5420032A (en) | 1991-12-23 | 1995-05-30 | Universitge Laval | Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5792632A (en) | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| CA2681922C (en) | 1994-01-18 | 2012-05-15 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| EP0752005B1 (en) | 1994-03-23 | 2008-10-08 | Ohio University | Compacted nucleic acids and their delivery to cells |
| US5585245A (en) | 1994-04-22 | 1996-12-17 | California Institute Of Technology | Ubiquitin-based split protein sensor |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| JP4118327B2 (ja) | 1994-08-20 | 2008-07-16 | ゲンダック・リミテッド | Dna認識のための結合タンパク質におけるまたはそれに関連する改良 |
| US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
| US5928638A (en) | 1996-06-17 | 1999-07-27 | Systemix, Inc. | Methods for gene transfer |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
| US6342345B1 (en) | 1997-04-02 | 2002-01-29 | The Board Of Trustees Of The Leland Stanford Junior University | Detection of molecular interactions by reporter subunit complementation |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| ATE466952T1 (de) | 1998-03-02 | 2010-05-15 | Massachusetts Inst Technology | Poly-zinkfinger-proteine mit verbesserten linkern |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| US20070060606A1 (en) * | 1999-10-07 | 2007-03-15 | Robertson Harold A | Compounds and methods for modulating phosphodiesterase 10A |
| DE60023936T2 (de) | 1999-12-06 | 2006-05-24 | Sangamo Biosciences Inc., Richmond | Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen |
| US6689558B2 (en) | 2000-02-08 | 2004-02-10 | Sangamo Biosciences, Inc. | Cells for drug discovery |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| ATE353361T1 (de) | 2000-04-28 | 2007-02-15 | Sangamo Biosciences Inc | Gezielten modifikation der chromatinstruktur |
| ATE374249T1 (de) | 2000-04-28 | 2007-10-15 | Sangamo Biosciences Inc | Datenbanken von regulatorischen sequenzen, methoden zu ihrer herstellung und verwendung |
| US20030044787A1 (en) | 2000-05-16 | 2003-03-06 | Joung J. Keith | Methods and compositions for interaction trap assays |
| US20040091878A1 (en) | 2000-07-21 | 2004-05-13 | Takashi Sera | Zinc finger domain recognition code and uses thereof |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| WO2002026959A2 (en) | 2000-09-28 | 2002-04-04 | Sangamo Biosciences, Inc. | Nuclear reprogramming using iwsi and related chromatin remodeling atpases |
| US6919204B2 (en) | 2000-09-29 | 2005-07-19 | Sangamo Biosciences, Inc. | Modulation of gene expression using localization domains |
| RU2192884C2 (ru) * | 2000-11-09 | 2002-11-20 | Государственное учреждение Научно-исследовательский институт клинической иммунологии СО РАМН | Вакцина для стимуляции противоопухолевого иммунитета |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| US20040224385A1 (en) | 2001-08-20 | 2004-11-11 | Barbas Carlos F | Zinc finger binding domains for cnn |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| EP1476547B1 (en) | 2002-01-23 | 2006-12-06 | The University of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| EP1485475B2 (en) | 2002-03-15 | 2017-09-20 | Cellectis | Hybrid and single chain meganucleases and use thereof |
| AU2003218382B2 (en) | 2002-03-21 | 2007-12-13 | Sangamo Therapeutics, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| US7074596B2 (en) | 2002-03-25 | 2006-07-11 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse mRNA cap analogues |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| EP1581610A4 (en) | 2002-09-05 | 2009-05-27 | California Inst Of Techn | USE OF CHIMERIC NUCLEASES TO STIMULATE GEN-TARGETING |
| WO2004067753A2 (en) | 2003-01-28 | 2004-08-12 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof. |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| DE602005011943D1 (de) | 2004-04-08 | 2009-02-05 | Sangamo Biosciences Inc | Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen |
| AU2005233550B2 (en) | 2004-04-08 | 2010-11-18 | Sangamo Therapeutics, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| US20060063231A1 (en) | 2004-09-16 | 2006-03-23 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| EP1877583A2 (en) | 2005-05-05 | 2008-01-16 | Arizona Board of Regents on behalf of the Unversity of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
| WO2007014275A2 (en) | 2005-07-26 | 2007-02-01 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| ES2602184T3 (es) | 2005-10-18 | 2017-02-20 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| US7985778B2 (en) | 2006-03-07 | 2011-07-26 | Koichi Nakamura | Method for decomposing and reclaiming synthetic resin having ester bond in the composition structure |
| AU2007254251A1 (en) | 2006-05-19 | 2007-11-29 | Sangamo Therapeutics, Inc. | Methods and compositions for inactivation of dihydrofolate reductase |
| JP5266210B2 (ja) | 2006-05-25 | 2013-08-21 | サンガモ バイオサイエンシズ インコーポレイテッド | 改変開裂ハーフドメイン |
| WO2007139982A2 (en) | 2006-05-25 | 2007-12-06 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
| DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| CN104072561B (zh) | 2007-06-19 | 2017-12-22 | 路易斯安那州州立大学及农业机械学院管理委员会 | 信使rna帽的抗‑反向硫代磷酸类似物的合成和用途 |
| US9506120B2 (en) | 2007-09-27 | 2016-11-29 | Sangamo Biosciences, Inc. | Rapid in vivo identification of biologically active nucleases |
| JP2010540534A (ja) | 2007-09-28 | 2010-12-24 | イントレキソン コーポレーション | 生体治療分子の発現のための治療遺伝子スイッチ構築物およびバイオリアクター、ならびにその使用 |
| EP2294191B1 (en) | 2008-06-10 | 2016-09-28 | Sangamo BioSciences, Inc. | Methods and compositions for generation of bax- and bak-deficient cell lines |
| US9206404B2 (en) | 2008-12-04 | 2015-12-08 | Sangamo Biosciences, Inc. | Method of deleting an IgM gene in an isolated rat cell |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| WO2011016840A2 (en) | 2009-07-28 | 2011-02-10 | Sangamo Biosciences, Inc. | Methods and compositions for treating trinucleotide repeat disorders |
| EP3165234B1 (de) | 2009-07-31 | 2019-04-03 | ethris GmbH | Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression |
| CA2788560A1 (en) | 2010-02-08 | 2011-08-11 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
| EP2563918B1 (en) | 2010-04-26 | 2016-11-23 | Sangamo BioSciences, Inc. | Genome editing of a rosa locus using zinc-finger nucleases |
| CN102959087B (zh) | 2010-05-03 | 2015-04-29 | 桑格摩生物科学股份有限公司 | 用于连接锌指模块的组合物 |
| CN103025344B (zh) | 2010-05-17 | 2016-06-29 | 桑格摩生物科学股份有限公司 | 新型dna-结合蛋白及其用途 |
| EP2441774B1 (en) | 2010-10-15 | 2018-12-12 | Fundació Centre de Regulació Genòmica | Peptides having zinc finger domains and uses thereof |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| EP3498833B1 (en) | 2011-09-21 | 2023-08-16 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| CA2852955C (en) | 2011-10-27 | 2021-02-16 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| CA2854819C (en) | 2011-11-16 | 2022-07-19 | Sangamo Biosciences, Inc. | Modified dna-binding proteins and uses thereof |
-
2014
- 2014-11-11 EP EP14860665.0A patent/EP3068905A4/en not_active Withdrawn
- 2014-11-11 US US15/036,020 patent/US10369201B2/en active Active
- 2014-11-11 RU RU2016123086A patent/RU2693891C1/ru active
- 2014-11-11 CA CA2929179A patent/CA2929179A1/en not_active Abandoned
- 2014-11-11 NZ NZ719477A patent/NZ719477A/en unknown
- 2014-11-11 CN CN201480072869.5A patent/CN105934524A/zh active Pending
- 2014-11-11 MX MX2016006106A patent/MX391904B/es unknown
- 2014-11-11 WO PCT/US2014/064987 patent/WO2015070212A1/en not_active Ceased
- 2014-11-11 JP JP2016528904A patent/JP2016537341A/ja active Pending
- 2014-11-11 KR KR1020167015301A patent/KR102431079B1/ko active Active
- 2014-11-11 BR BR112016010175A patent/BR112016010175A2/pt active Search and Examination
- 2014-11-11 AU AU2014346424A patent/AU2014346424B2/en active Active
-
2019
- 2019-03-29 JP JP2019067000A patent/JP2019116502A/ja active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010530239A (ja) * | 2007-06-18 | 2010-09-09 | コミッサリア ア レネルジ アトミック | 変異および内因性野生型ハンチンチン遺伝子のsiRNAによる可逆的サイレンシングおよびハンチントン病の治療のためのその応用 |
| WO2013130824A1 (en) * | 2012-02-29 | 2013-09-06 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
Non-Patent Citations (2)
| Title |
|---|
| NEUROSCIENCE, vol. 123, JPN6018018594, 2004, pages 967 - 981, ISSN: 0004035640 * |
| PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 95, JPN6018018595, 1998, pages 6480 - 6485, ISSN: 0004035641 * |
Cited By (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200077529A (ko) * | 2017-10-24 | 2020-06-30 | 상가모 테라퓨틱스, 인코포레이티드 | 희귀 질환의 치료를 위한 방법 및 조성물 |
| JP2021500079A (ja) * | 2017-10-24 | 2021-01-07 | サンガモ セラピューティクス, インコーポレイテッド | 希少疾患の処置のための方法および組成物 |
| JP7381476B2 (ja) | 2017-10-24 | 2023-11-15 | サンガモ セラピューティクス, インコーポレイテッド | 希少疾患の処置のための方法および組成物 |
| KR102705509B1 (ko) | 2017-10-24 | 2024-09-12 | 상가모 테라퓨틱스, 인코포레이티드 | 희귀 질환의 치료를 위한 방법 및 조성물 |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112016010175A2 (pt) | 2017-10-03 |
| CN105934524A (zh) | 2016-09-07 |
| US20160296605A1 (en) | 2016-10-13 |
| KR102431079B1 (ko) | 2022-08-11 |
| RU2693891C1 (ru) | 2019-07-05 |
| RU2016123086A (ru) | 2017-12-19 |
| CA2929179A1 (en) | 2015-05-14 |
| KR20160077208A (ko) | 2016-07-01 |
| AU2014346424A1 (en) | 2016-05-19 |
| US10369201B2 (en) | 2019-08-06 |
| EP3068905A4 (en) | 2017-07-05 |
| EP3068905A1 (en) | 2016-09-21 |
| NZ719477A (en) | 2022-05-27 |
| JP2019116502A (ja) | 2019-07-18 |
| MX391904B (es) | 2025-03-12 |
| AU2014346424B2 (en) | 2020-09-17 |
| WO2015070212A1 (en) | 2015-05-14 |
| MX2016006106A (es) | 2016-12-09 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US11723952B2 (en) | Methods and compositions for treating Huntington's Disease | |
| RU2693891C1 (ru) | Способы и композиции для лечения болезни хантингтона | |
| JP6290853B2 (ja) | トリヌクレオチド反復疾患を治療するための方法および組成物 | |
| US12043650B2 (en) | Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (CFTR) gene |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20170904 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20170904 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20180523 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20180817 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20181204 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20190329 |
|
| C60 | Trial request (containing other claim documents, opposition documents) |
Free format text: JAPANESE INTERMEDIATE CODE: C60 Effective date: 20190329 |
|
| A911 | Transfer to examiner for re-examination before appeal (zenchi) |
Free format text: JAPANESE INTERMEDIATE CODE: A911 Effective date: 20190416 |
|
| C21 | Notice of transfer of a case for reconsideration by examiners before appeal proceedings |
Free format text: JAPANESE INTERMEDIATE CODE: C21 Effective date: 20190417 |
|
| A912 | Re-examination (zenchi) completed and case transferred to appeal board |
Free format text: JAPANESE INTERMEDIATE CODE: A912 Effective date: 20190517 |
|
| C211 | Notice of termination of reconsideration by examiners before appeal proceedings |
Free format text: JAPANESE INTERMEDIATE CODE: C211 Effective date: 20190521 |
|
| C22 | Notice of designation (change) of administrative judge |
Free format text: JAPANESE INTERMEDIATE CODE: C22 Effective date: 20191205 |
|
| C22 | Notice of designation (change) of administrative judge |
Free format text: JAPANESE INTERMEDIATE CODE: C22 Effective date: 20200403 |
|
| C13 | Notice of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: C13 Effective date: 20200603 |
|
| C28A | Non-patent document cited |
Free format text: JAPANESE INTERMEDIATE CODE: C2838 Effective date: 20200603 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20200831 |
|
| C23 | Notice of termination of proceedings |
Free format text: JAPANESE INTERMEDIATE CODE: C23 Effective date: 20201005 |
|
| C03 | Trial/appeal decision taken |
Free format text: JAPANESE INTERMEDIATE CODE: C03 Effective date: 20201106 |
|
| C30A | Notification sent |
Free format text: JAPANESE INTERMEDIATE CODE: C3012 Effective date: 20201106 |