MX374399B - Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. - Google Patents
Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.Info
- Publication number
- MX374399B MX374399B MX2018009444A MX2018009444A MX374399B MX 374399 B MX374399 B MX 374399B MX 2018009444 A MX2018009444 A MX 2018009444A MX 2018009444 A MX2018009444 A MX 2018009444A MX 374399 B MX374399 B MX 374399B
- Authority
- MX
- Mexico
- Prior art keywords
- disease
- compositions
- treating
- preventing huntington
- huntington
- Prior art date
Links
- 208000023105 Huntington disease Diseases 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
-
- 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
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4703—Inhibitors; Suppressors
-
- 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
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
-
- 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
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Plant Pathology (AREA)
- Marine Sciences & Fisheries (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Neurology (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se describen composiciones y sus usos para tratar y prevenir la enfermedad de Huntington.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261605028P | 2012-02-29 | 2012-02-29 | |
| PCT/US2013/028348 WO2013130824A1 (en) | 2012-02-29 | 2013-02-28 | Methods and compositions for treating huntington's disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX374399B true MX374399B (es) | 2025-03-06 |
Family
ID=49083290
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2014010455A MX359327B (es) | 2012-02-29 | 2013-02-28 | Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. |
| MX2018009444A MX374399B (es) | 2012-02-29 | 2013-02-28 | Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2014010455A MX359327B (es) | 2012-02-29 | 2013-02-28 | Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. |
Country Status (14)
| Country | Link |
|---|---|
| US (7) | US8841260B2 (es) |
| EP (1) | EP2820159B1 (es) |
| JP (3) | JP6490426B2 (es) |
| KR (1) | KR102084539B1 (es) |
| CN (2) | CN108285491B (es) |
| AU (1) | AU2013225950B2 (es) |
| BR (1) | BR112014021104B1 (es) |
| CA (1) | CA2865011C (es) |
| HK (1) | HK1203566A1 (es) |
| MX (2) | MX359327B (es) |
| NZ (1) | NZ629427A (es) |
| RU (1) | RU2639277C2 (es) |
| WO (1) | WO2013130824A1 (es) |
| ZA (1) | ZA201406209B (es) |
Families Citing this family (183)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| EP2766498B1 (en) | 2011-10-14 | 2019-06-19 | President and Fellows of Harvard College | Sequencing by structure assembly |
| ES2991004T3 (es) | 2011-12-22 | 2024-12-02 | Harvard College | Métodos para la detección de analitos |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| AU2013225950B2 (en) | 2012-02-29 | 2018-02-15 | Sangamo Therapeutics, Inc. | Methods and compositions for treating huntington's disease |
| US20160046961A1 (en) | 2012-05-25 | 2016-02-18 | Emmanuelle Charpentier | Methods and Compositions for RNA-Directed Target DNA Modification and For RNA-Directed Modulation of Transcription |
| EP2928496B1 (en) | 2012-12-06 | 2019-10-09 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| CA2894668A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells |
| JP2016501531A (ja) | 2012-12-12 | 2016-01-21 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作および治療適用のための系、方法および組成物の送達、エンジニアリングおよび最適化 |
| ES2701749T3 (es) | 2012-12-12 | 2019-02-25 | Broad Inst Inc | Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos |
| US9970024B2 (en) | 2012-12-17 | 2018-05-15 | President And Fellows Of Harvard College | RNA-guided human genome engineering |
| US10138509B2 (en) | 2013-03-12 | 2018-11-27 | President And Fellows Of Harvard College | Method for generating a three-dimensional nucleic acid containing matrix |
| WO2014159247A1 (en) | 2013-03-14 | 2014-10-02 | University Of Florida Research Foundation, Inc. | Di-amino acid repeat-containing proteins associated with als |
| ES2901396T3 (es) | 2013-03-14 | 2022-03-22 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| US11332719B2 (en) * | 2013-03-15 | 2022-05-17 | The Broad Institute, Inc. | Recombinant virus and preparations thereof |
| EP2971041B1 (en) | 2013-03-15 | 2018-11-28 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
| EP4286517A3 (en) | 2013-04-04 | 2024-03-13 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| US9267135B2 (en) | 2013-06-04 | 2016-02-23 | President And Fellows Of Harvard College | RNA-guided transcriptional regulation |
| MY197877A (en) | 2013-06-04 | 2023-07-22 | Harvard College | Rna-guided transcriptional regulation |
| EP3825406A1 (en) | 2013-06-17 | 2021-05-26 | The Broad Institute Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
| EP3011030B1 (en) | 2013-06-17 | 2023-11-08 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| EP3620524A1 (en) | 2013-06-17 | 2020-03-11 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
| SG10201710487VA (en) | 2013-06-17 | 2018-01-30 | Broad Inst Inc | Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Viral Components |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| US10011850B2 (en) | 2013-06-21 | 2018-07-03 | The General Hospital Corporation | Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing |
| EP3019595A4 (en) | 2013-07-09 | 2016-11-30 | THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS | |
| RU2748433C2 (ru) | 2013-07-10 | 2021-05-25 | Президент Энд Фэллоуз Оф Харвард Коллидж | ОРТОГОНАЛЬНЫЕ БЕЛКИ Cas9 ДЛЯ РНК-НАПРАВЛЯЕМОЙ РЕГУЛЯЦИИ И РЕДАКТИРОВАНИЯ ГЕНОВ |
| US11306328B2 (en) | 2013-07-26 | 2022-04-19 | President And Fellows Of Harvard College | Genome engineering |
| 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 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| WO2015059265A1 (en) | 2013-10-25 | 2015-04-30 | Cellectis | Design of rare-cutting endonucleases for efficient and specific targeting dna sequences comprising highly repetitive motives |
| WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
| KR102523466B1 (ko) | 2013-11-07 | 2023-04-20 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| MX391904B (es) * | 2013-11-11 | 2025-03-12 | Sangamo Biosciences Inc | Un represor genetico para usarse en el tratamiento de la enfermedad de huntington. |
| US9074199B1 (en) | 2013-11-19 | 2015-07-07 | President And Fellows Of Harvard College | Mutant Cas9 proteins |
| US10787684B2 (en) | 2013-11-19 | 2020-09-29 | President And Fellows Of Harvard College | Large gene excision and insertion |
| EP2878667A1 (en) * | 2013-11-29 | 2015-06-03 | Institut Pasteur | TAL effector means useful for partial or full deletion of DNA tandem repeats |
| BR112016013547A2 (pt) * | 2013-12-12 | 2017-10-03 | Broad Inst Inc | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
| WO2015089462A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| JP2017503485A (ja) | 2013-12-12 | 2017-02-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 遺伝子産物の発現、構造情報、及び誘導性モジュラーcas酵素を変更するためのcrispr−cas系並びに方法 |
| EP4219699A1 (en) | 2013-12-12 | 2023-08-02 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| EP3470089A1 (en) | 2013-12-12 | 2019-04-17 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| KR20160089530A (ko) | 2013-12-12 | 2016-07-27 | 더 브로드 인스티튜트, 인코퍼레이티드 | Hbv 및 바이러스 질병 및 질환을 위한 crisprcas 시스템 및 조성물의 전달,용도 및 치료적 적용 |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| EP3105325B2 (en) | 2014-02-13 | 2024-10-23 | Takara Bio USA, Inc. | Methods of depleting a target molecule from an initial collection of nucleic acids, and compositions and kits for practicing the same |
| WO2015134812A1 (en) | 2014-03-05 | 2015-09-11 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US9938521B2 (en) | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| EP3929279A1 (en) * | 2014-03-18 | 2021-12-29 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of zinc finger protein expression |
| US11242525B2 (en) | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| CN106413760B (zh) * | 2014-05-08 | 2020-01-14 | 桑格摩生物科学股份有限公司 | 用于治疗亨廷顿病的方法和组合物 |
| WO2015175642A2 (en) * | 2014-05-13 | 2015-11-19 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
| WO2015188056A1 (en) | 2014-06-05 | 2015-12-10 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease design |
| US10179932B2 (en) | 2014-07-11 | 2019-01-15 | President And Fellows Of Harvard College | Methods for high-throughput labelling and detection of biological features in situ using microscopy |
| WO2016019144A2 (en) | 2014-07-30 | 2016-02-04 | Sangamo Biosciences, Inc. | Gene correction of scid-related genes in hematopoietic stem and progenitor cells |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| EP2982758A1 (en) | 2014-08-04 | 2016-02-10 | Centre Hospitalier Universitaire Vaudois (CHUV) | Genome editing for the treatment of huntington's disease |
| ES2730378T3 (es) | 2014-08-27 | 2019-11-11 | Caribou Biosciences Inc | Procedimientos para incrementar la eficiencia de la modificación mediada por Cas9 |
| EP3201340B1 (en) | 2014-10-01 | 2020-12-02 | The General Hospital Corporation | Methods for increasing efficiency of nuclease-induced homology-directed repair |
| GB201418965D0 (es) | 2014-10-24 | 2014-12-10 | Ospedale San Raffaele And Fond Telethon | |
| EP3215623A4 (en) | 2014-11-06 | 2018-09-26 | President and Fellows of Harvard College | Cells lacking b2m surface expression and methods for allogeneic administration of such cells |
| CA2975583A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| EP3985115A1 (en) | 2014-12-12 | 2022-04-20 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| AU2015369725A1 (en) | 2014-12-24 | 2017-06-29 | Massachusetts Institute Of Technology | CRISPR having or associated with destabilization domains |
| PE20171382A1 (es) | 2015-02-10 | 2017-09-15 | Genzyme Corp | ARNi VARIANTE |
| CA2982966C (en) | 2015-04-24 | 2024-02-20 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| US10179918B2 (en) | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
| JP2018515139A (ja) | 2015-05-08 | 2018-06-14 | 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 | 万能ドナー幹細胞および関連する方法 |
| WO2016196324A1 (en) * | 2015-05-29 | 2016-12-08 | University Of Floridia Research Foundation, Inc. | Methods for diagnosing huntington's disease |
| RU2752834C2 (ru) | 2015-06-18 | 2021-08-09 | Те Брод Инститьют, Инк. | Мутации фермента crispr, уменьшающие нецелевые эффекты |
| CA3012607A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzymes and systems |
| WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
| US9580727B1 (en) | 2015-08-07 | 2017-02-28 | Caribou Biosciences, Inc. | Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides |
| US9926546B2 (en) | 2015-08-28 | 2018-03-27 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| MX391052B (es) * | 2015-09-23 | 2025-03-21 | Sangamo Therapeutics Inc | Represores de htt y usos de estos. |
| DK3350327T3 (en) | 2015-10-23 | 2019-01-21 | Caribou Biosciences Inc | CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID |
| CA3002827A1 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| EP3371329A4 (en) | 2015-11-03 | 2019-06-19 | President and Fellows of Harvard College | METHOD AND DEVICE FOR VOLUMETRIC IMAGING OF A THREE-DIMENSIONAL NUCLEIC ACID-CONTAINING MATRIX |
| EP3881857B1 (en) | 2016-02-18 | 2025-04-16 | The Penn State Research Foundation | Generating gabaergic neurons in brains |
| WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| EP3440100A4 (en) | 2016-04-04 | 2020-01-22 | University of Florida Research Foundation, Incorporated | HANDLING EIF3 TO MODULATE A NON-ATG TRANSLATION ASSOCIATED WITH A REPETITION (RAN) |
| CN109415761B (zh) | 2016-04-25 | 2022-09-20 | 哈佛学院董事及会员团体 | 用于原位分子检测的杂交链反应方法 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| KR20220108216A (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| CN109863143B (zh) | 2016-07-13 | 2021-10-15 | 威泰克斯制药公司 | 提高基因组编辑效率的方法、组合物和试剂盒 |
| CA3032822A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| KR102827276B1 (ko) | 2016-08-03 | 2025-07-01 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| JP7201153B2 (ja) | 2016-08-09 | 2023-01-10 | 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 | プログラム可能cas9-リコンビナーゼ融合タンパク質およびその使用 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| CA3035534A1 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| IL266862B2 (en) | 2016-12-01 | 2024-01-01 | Sangamo Therapeutics Inc | Tau modulators and methods and preparations for their administration |
| US9816093B1 (en) | 2016-12-06 | 2017-11-14 | Caribou Biosciences, Inc. | Engineered nucleic acid-targeting nucleic acids |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| US11268093B2 (en) | 2017-01-03 | 2022-03-08 | Rula Zain-Luqman | Therapeutic method for huntington's disease |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| WO2018165629A1 (en) | 2017-03-10 | 2018-09-13 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| JP7526455B2 (ja) | 2017-04-17 | 2024-08-01 | ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. | PKRおよびeIF2A-P経路によるRANタンパク質翻訳の調節 |
| BR112019021977A2 (pt) | 2017-04-20 | 2020-06-16 | Egenesis, Inc. | Métodos para a geração de animais geneticamente modificados |
| SG11201909868YA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| JP2020530979A (ja) | 2017-06-30 | 2020-11-05 | インスクリプタ, インコーポレイテッド | 自動細胞処理方法、モジュール、機器及びシステム |
| 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 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| US11603529B2 (en) | 2017-09-22 | 2023-03-14 | Genzyme Corporation | Variant RNAi |
| AU2018342105B2 (en) | 2017-09-26 | 2023-11-16 | University Of Florida Research Foundation, Incorporated | Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders |
| CA3074927A1 (en) | 2017-09-30 | 2019-04-04 | Inscripta, Inc. | Flow through electroporation instrumentation |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| US12121524B2 (en) | 2018-01-17 | 2024-10-22 | Vertex Pharmaceuticals Incorporated | DNA-PK inhibitors |
| IL276080B2 (en) | 2018-01-17 | 2023-10-01 | Vertex Pharma | Dna-pk inhibitor compounds, compositions comprising same and uses thereof |
| EP3740483B1 (en) | 2018-01-17 | 2023-01-11 | Vertex Pharmaceuticals Incorporated | Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency |
| US20190233816A1 (en) | 2018-01-26 | 2019-08-01 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| MX2020009487A (es) | 2018-03-14 | 2020-12-10 | Editas Medicine Inc | Sistemas y metodos para el tratamiento de hemoglobinopatias. |
| JP7332622B2 (ja) | 2018-04-18 | 2023-08-23 | サンガモ セラピューティクス, インコーポレイテッド | ハンチンチン(htt)の調節のためのジンクフィンガータンパク質組成物 |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| CN108728491B (zh) * | 2018-06-21 | 2022-08-26 | 陕西杆粒生物科技有限公司 | 一种广谱抗凋亡杆状病毒表达载体 |
| EP3814493A4 (en) * | 2018-06-27 | 2022-03-30 | Altius Institute For Biomedical Sciences | NUCLEIC ACID BINDING DOMAINS AND METHODS OF USE THEREOF |
| AR114540A1 (es) | 2018-08-03 | 2020-09-16 | Genzyme Corp | VARIANTE DE ARNi CONTRA a-SINUCLEÍNA |
| US12421507B2 (en) | 2018-08-20 | 2025-09-23 | The Broad Institute, Inc. | Methods and compositions for optochemical control of CRISPR-CAS9 |
| WO2020072677A1 (en) | 2018-10-02 | 2020-04-09 | Sangamo Therapeutics, Inc. | Methods and compositions for modulation of tau proteins |
| CN113195002A (zh) * | 2018-10-02 | 2021-07-30 | 桑格摩生物治疗股份有限公司 | 工程化的遗传调节物 |
| CA3114699A1 (en) | 2018-10-09 | 2020-04-16 | The University Of British Columbia | Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto |
| WO2020076976A1 (en) | 2018-10-10 | 2020-04-16 | Readcoor, Inc. | Three-dimensional spatial molecular indexing |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US12264323B2 (en) | 2018-12-17 | 2025-04-01 | The Broad Institute, Inc. | CRISPR CPF1 direct repeat variants |
| EP3898958A1 (en) | 2018-12-17 | 2021-10-27 | The Broad Institute, Inc. | Crispr-associated transposase systems and methods of use 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 |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst 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 |
| PE20212332A1 (es) | 2019-04-23 | 2021-12-14 | Sangamo Therapeutics Inc | Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos |
| BR112021026381A2 (pt) * | 2019-06-28 | 2022-02-08 | Penn State Res Found | Métodos e materiais para tratamento da doença de huntington |
| US20220267732A1 (en) | 2019-08-01 | 2022-08-25 | Sana Biotechnology, Inc. | Dux4 expressing cells and uses thereof |
| JP7728747B2 (ja) | 2019-08-23 | 2025-08-25 | サナ バイオテクノロジー,インコーポレイテッド | Cd24発現細胞およびそれらの用途 |
| EP4031562A4 (en) | 2019-09-20 | 2023-11-01 | University of Florida Research Foundation, Incorporated | DETECTION OF ANTIBODIES AGAINST RAN PROTEINS FROM SERUM AND TISSUE LYSATES |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| 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 |
| KR20220094213A (ko) * | 2019-11-01 | 2022-07-05 | 알닐람 파마슈티칼스 인코포레이티드 | 헌팅틴 (HTT) iRNA 제제 조성물 및 이의 사용 방법 |
| US20230079440A1 (en) | 2019-11-01 | 2023-03-16 | Sangamo Therapeutics, Inc. | Compositions and methods for genome engineering |
| JP2023520997A (ja) | 2020-03-25 | 2023-05-23 | サナ バイオテクノロジー,インコーポレイテッド | 神経学的障害及び神経学的病態の処置のための低免疫原性神経細胞 |
| EP4146804A1 (en) | 2020-05-08 | 2023-03-15 | The Broad Institute Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2021236852A1 (en) | 2020-05-20 | 2021-11-25 | Sana Biotechnology, Inc. | Methods and compositions for treatment of viral infections |
| WO2022036150A1 (en) | 2020-08-13 | 2022-02-17 | Sana Biotechnology, Inc. | Methods of treating sensitized patients with hypoimmunogenic cells, and associated methods and compositions |
| US20220049303A1 (en) | 2020-08-17 | 2022-02-17 | Readcoor, Llc | Methods and systems for spatial mapping of genetic variants |
| RU2748997C1 (ru) * | 2020-12-11 | 2021-06-02 | Федеральное государственное автономное образовательное учреждение высшего образования "Российский национальный исследовательский медицинский университет имени Н.И. Пирогова" Министерства здравоохранения Российской Федерации (ФГАОУ ВО РНИМУ им. Н.И. Пирогова Минздрава России) | Универсальный эпигеномный репрессор транскрипции целевых генов в клетках человека |
| IL303473A (en) | 2020-12-31 | 2023-08-01 | Sana Biotechnology Inc | Methods and compositions for modulating car-t activity |
| BR112023024434A2 (pt) | 2021-05-27 | 2024-02-20 | Sana Biotechnology Inc | Células hipoimunogênicas que compreendem hla-e ou hla-g geneticamente modificadas |
| JP2024530403A (ja) | 2021-07-14 | 2024-08-21 | サナ バイオテクノロジー,インコーポレイテッド | 低免疫原性細胞におけるy染色体連鎖抗原の変化した発現 |
| AU2022326565A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy |
| WO2023019229A1 (en) | 2021-08-11 | 2023-02-16 | Sana Biotechnology, Inc. | Genetically modified primary cells for allogeneic cell therapy |
| KR20240053673A (ko) | 2021-08-11 | 2024-04-24 | 사나 바이오테크놀로지, 인크. | 저면역원성 세포 내 유전자 발현을 변경하기 위한 유도성 시스템 |
| WO2023019225A2 (en) | 2021-08-11 | 2023-02-16 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions |
| AU2022325231A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions |
| WO2023069790A1 (en) | 2021-10-22 | 2023-04-27 | Sana Biotechnology, Inc. | Methods of engineering allogeneic t cells with a transgene in a tcr locus and associated compositions and methods |
| WO2023122337A1 (en) | 2021-12-23 | 2023-06-29 | Sana Biotechnology, Inc. | Chimeric antigen receptor (car) t cells for treating autoimmune disease and associated methods |
| WO2023154578A1 (en) | 2022-02-14 | 2023-08-17 | Sana Biotechnology, Inc. | Methods of treating patients exhibiting a prior failed therapy with hypoimmunogenic cells |
| KR20240155390A (ko) | 2022-02-17 | 2024-10-28 | 사나 바이오테크놀로지, 인크. | 조작된 cd47 단백질 및 이의 용도 |
| WO2023173123A1 (en) | 2022-03-11 | 2023-09-14 | Sana Biotechnology, Inc. | Genetically modified cells and compositions and uses thereof |
| 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 |
| WO2024151541A1 (en) | 2023-01-09 | 2024-07-18 | Sana Biotechnology, Inc. | Type-1 diabetes autoimmune mouse |
| AU2024264889A1 (en) | 2023-05-03 | 2025-11-13 | Sana Biotechnology, Inc. | Methods of dosing and administration of engineered islet cells |
| WO2024243236A2 (en) | 2023-05-22 | 2024-11-28 | Sana Biotechnology, Inc. | Methods of delivery of islet cells and related methods |
| WO2025004001A1 (en) | 2023-06-30 | 2025-01-02 | Takeda Pharmaceutical Company Limited | Htt repressors and uses thereof |
| WO2025106739A1 (en) * | 2023-11-14 | 2025-05-22 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of htt expression |
Family Cites Families (102)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US789538A (en) | 1904-11-11 | 1905-05-09 | Colin E Ham | Dumb-bell. |
| 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 |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of 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 |
| DE69534629D1 (de) | 1994-01-18 | 2005-12-29 | Scripps Research Inst | Derivate von zinkfingerproteinen und methoden |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | 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 |
| 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 |
| ATE524545T1 (de) | 1994-08-20 | 2011-09-15 | Gendaq Ltd | Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von dna |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| 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 |
| ES2341926T3 (es) | 1998-03-02 | 2010-06-29 | Massachusetts Institute Of Technology | Poliproteinas con dedos de cinc que tienen enlazadores mejorados. |
| 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 |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| 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 |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| 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 |
| CA2394850C (en) | 1999-12-06 | 2012-02-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| EP2207032A1 (en) | 2000-02-08 | 2010-07-14 | Sangamo BioSciences, Inc. | Cells expressing zinc finger protein for drug discovery |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| US7001768B2 (en) | 2000-04-28 | 2006-02-21 | Sangamo Biosciences, Inc. | Targeted modification of chromatin structure |
| US7217509B2 (en) | 2000-04-28 | 2007-05-15 | Sangamo Biosciences, Inc. | Databases of regulatory sequences; methods of making and using same |
| AU2001263155A1 (en) | 2000-05-16 | 2001-11-26 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| EP1303608A2 (en) * | 2000-07-21 | 2003-04-23 | Syngenta Participations AG | 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 |
| US6919204B2 (en) | 2000-09-29 | 2005-07-19 | Sangamo Biosciences, Inc. | Modulation of gene expression using localization domains |
| AU2002217929A1 (en) | 2000-11-28 | 2002-06-11 | Sangamo Biosciences, Inc. | Modulation of gene expression using insulator binding proteins |
| 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 |
| AU2002336373A1 (en) | 2001-08-20 | 2003-03-03 | The Scripps Research Institute | 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 |
| WO2003087341A2 (en) | 2002-01-23 | 2003-10-23 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| AU2003215869B2 (en) | 2002-03-15 | 2008-04-24 | Cellectis | Hybrid and single chain meganucleases and use thereof |
| US20030232410A1 (en) | 2002-03-21 | 2003-12-18 | Monika Liljedahl | 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 |
| US9447434B2 (en) | 2002-09-05 | 2016-09-20 | California Institute Of Technology | Use of chimeric nucleases to stimulate gene targeting |
| JP2006518372A (ja) | 2003-01-28 | 2006-08-10 | セレクティス | 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用 |
| 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 |
| KR100535326B1 (ko) * | 2004-01-20 | 2005-12-09 | 한국생명공학연구원 | 줄기 세포로부터 자연살해 세포로의 분화 조절용 유전자를유효성분으로 포함하는 분화 조절제 |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| ATE418346T1 (de) | 2004-04-08 | 2009-01-15 | Sangamo Biosciences Inc | Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen |
| WO2005100392A2 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| CA2579677A1 (en) | 2004-09-16 | 2006-03-30 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| US7358085B2 (en) | 2005-02-28 | 2008-04-15 | Sangamo Biosciences, Inc. | Anti-angiogenic methods and compositions |
| 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 |
| AU2006272634B2 (en) | 2005-07-26 | 2013-01-24 | Sangamo Therapeutics, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| EP2662442B1 (en) | 2005-10-18 | 2015-03-25 | Precision Biosciences | Rationally designed meganuclease with altered dimer formation affinity. |
| EP1941059A4 (en) * | 2005-10-28 | 2010-11-03 | Alnylam Pharmaceuticals Inc | COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF THE HUNTINGTIN GENE |
| JP2009537140A (ja) | 2006-05-19 | 2009-10-29 | サンガモ バイオサイエンシーズ, インコーポレイテッド | ジヒドロ葉酸還元酵素の不活性化のための方法及び組成物 |
| DE602007005634D1 (de) | 2006-05-25 | 2010-05-12 | Sangamo Biosciences Inc | Variante foki-spaltungshälften-domänen |
| ATE536371T1 (de) | 2006-05-25 | 2011-12-15 | Sangamo Biosciences Inc | Verfahren und zusammensetzungen zur gendeaktivierung |
| NZ597995A (en) | 2006-12-14 | 2013-05-31 | Dow Agrosciences Llc | Optimized non-canonical zinc finger proteins |
| UA100505C2 (ru) * | 2006-12-14 | 2013-01-10 | ДАУ АГРОСАЙЕНСИЗ ЕлЕлСи | Белок с цинковыми пальцами |
| DE602007005629D1 (de) | 2007-06-18 | 2010-05-12 | Commissariat Energie Atomique | Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington |
| DK2167523T3 (da) | 2007-06-19 | 2014-09-08 | Univ Louisiana State | Syntese og anvendelse af anti-reverse phosphorothioatanaloger af messenger-rna-cappen |
| WO2009042163A2 (en) | 2007-09-27 | 2009-04-02 | Sangamo Biosciences, Inc. | Rapid in vivo identification of biologically active nucleases |
| EP2205249B1 (en) | 2007-09-28 | 2018-11-07 | Intrexon Corporation | Therapeutic gene-switch constructs and bioreactors for the expression of biotherapeutic molecules, and uses thereof |
| DE102007056956B4 (de) | 2007-11-27 | 2009-10-29 | Moosbauer, Peter, Dipl.-Ing.(FH) | Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung |
| JP5763530B2 (ja) | 2008-06-10 | 2015-08-12 | サンガモ バイオサイエンシーズ, インコーポレイテッド | Bax−およびBak−欠損細胞株の生成のための方法および組成物 |
| 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 |
| EP2534173B1 (en) | 2010-02-08 | 2019-09-11 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| CA2797189C (en) | 2010-05-03 | 2020-05-26 | Sangamo Biosciences, Inc. | Compositions for linking zinc finger modules |
| AU2011256838B2 (en) | 2010-05-17 | 2014-10-09 | Sangamo Therapeutics, Inc. | Novel DNA-binding proteins and uses thereof |
| ES2713560T3 (es) * | 2010-10-15 | 2019-05-22 | Fund Centre De Regulacio Genòmica | Péptidos que tienen dominios de dedos de zinc y usos de estos |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| AU2013225950B2 (en) | 2012-02-29 | 2018-02-15 | Sangamo Therapeutics, Inc. | Methods and compositions for treating huntington's disease |
-
2013
- 2013-02-28 AU AU2013225950A patent/AU2013225950B2/en active Active
- 2013-02-28 BR BR112014021104-3A patent/BR112014021104B1/pt active IP Right Grant
- 2013-02-28 CN CN201810089961.2A patent/CN108285491B/zh active Active
- 2013-02-28 RU RU2014138989A patent/RU2639277C2/ru active
- 2013-02-28 MX MX2014010455A patent/MX359327B/es active IP Right Grant
- 2013-02-28 NZ NZ629427A patent/NZ629427A/en unknown
- 2013-02-28 CN CN201380016072.9A patent/CN104204225A/zh active Pending
- 2013-02-28 MX MX2018009444A patent/MX374399B/es unknown
- 2013-02-28 HK HK15103974.7A patent/HK1203566A1/xx unknown
- 2013-02-28 US US13/781,176 patent/US8841260B2/en active Active
- 2013-02-28 KR KR1020147026364A patent/KR102084539B1/ko active Active
- 2013-02-28 CA CA2865011A patent/CA2865011C/en active Active
- 2013-02-28 EP EP13755872.2A patent/EP2820159B1/en active Active
- 2013-02-28 WO PCT/US2013/028348 patent/WO2013130824A1/en not_active Ceased
- 2013-02-28 JP JP2014560044A patent/JP6490426B2/ja active Active
-
2014
- 2014-07-29 US US14/446,189 patent/US9499597B2/en active Active
- 2014-08-22 ZA ZA2014/06209A patent/ZA201406209B/en unknown
-
2016
- 2016-10-14 US US15/294,062 patent/US10265377B2/en active Active
-
2017
- 2017-05-23 JP JP2017101508A patent/JP2017143835A/ja active Pending
-
2018
- 2018-11-19 JP JP2018216307A patent/JP2019022531A/ja active Pending
-
2019
- 2019-03-04 US US16/291,708 patent/US10471123B2/en active Active
- 2019-09-30 US US16/587,428 patent/US10857203B2/en active Active
-
2020
- 2020-11-16 US US17/098,970 patent/US11723952B2/en active Active
-
2023
- 2023-06-29 US US18/344,757 patent/US20230414710A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MX374399B (es) | Composiciones y sus usos para tratar y prevenir la enfermedad de huntington. | |
| MX2015003140A (es) | Formulaciones de enzalutamida. | |
| BR112016026378A2 (pt) | formulações e métodos para tratamento com queratina | |
| MX2017013562A (es) | Reguladores de microbioma y usos relacionados de los mismos. | |
| EP3547968A4 (en) | PROSTHESIS OBTAINED BY TISSUE ENGINEERING | |
| MX358514B (es) | Compuestos para tratar la atrofia muscular espinal. | |
| TR201813877T4 (tr) | Spi̇nal müsküler atrofi̇ni̇n tedavi̇ edi̇lmesi̇ne yöneli̇k bi̇leşi̇kler. | |
| UY34484A (es) | Benzotienilo-pirrolotriazinas disustituidas y sus usos | |
| UY34750A (es) | ?compuestos para el tratamiento del hiv, composiciones,métodos de preparación, intermediarios y métodos terapéuticos?. | |
| AR091959A1 (es) | Agentes de sosten sinteticos y agentes de sosten monodispersos y metodos para la elaboracion de los mismos | |
| UY35427A (es) | Compuestos y sus usos en la modulación de la hemoglobina | |
| MX379016B (es) | Composiciones y metodos para tratar enfermedad de gaucher. | |
| UY34210A (es) | Composiciones, métodos y sistemas para la micropropagación de plantas | |
| DK3010506T3 (da) | Pridopidin i behandling af huntingtons sygdom | |
| UY35425A (es) | Compuestos y sus usos en la modulación de la hemoglobina | |
| UY35426A (es) | Compuestos y sus usos en la modulación de la hemoglobina | |
| CR20150047A (es) | Anticuerpos anti-cd22 e inmunoconjugados | |
| UY34165A (es) | ?compuestos aldh-2 mitocondriales humanos para el tratamiento de la adicción?. | |
| MX359769B (es) | Metodos para descelularizar huesos. | |
| UY34636A (es) | Composiciones tópicas que comprenden fipronilo y permetrina y sus métodos de uso | |
| MX2016009590A (es) | Composiciones de apilimod y métodos para usar las mismas. | |
| UY34725A (es) | Nuevos derivados de tienopirimidina, procesos para su preparación y sus usos terapéuticos. | |
| MX2015003701A (es) | Composiciones para tratamiento. | |
| BR112014031512A2 (pt) | composição cosmética e método para o tratamento cosmético. | |
| BR112015027841A2 (pt) | Método para umedecimento e/ou condicionamento do solo, concentrado da composição para tratamento do solo, e, uso de uma composição para tratamento do solo |