JPWO2018102665A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2018102665A5 JPWO2018102665A5 JP2019529263A JP2019529263A JPWO2018102665A5 JP WO2018102665 A5 JPWO2018102665 A5 JP WO2018102665A5 JP 2019529263 A JP2019529263 A JP 2019529263A JP 2019529263 A JP2019529263 A JP 2019529263A JP WO2018102665 A5 JPWO2018102665 A5 JP WO2018102665A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- gene
- dna binding
- modulator
- binding domains
- 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.)
- Granted
Links
- 108090000623 proteins and genes Proteins 0.000 claims description 58
- 101710185494 Zinc finger protein Proteins 0.000 claims description 28
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 28
- 230000004568 DNA-binding Effects 0.000 claims description 26
- 239000008194 pharmaceutical composition Substances 0.000 claims description 21
- 108091033319 polynucleotide Proteins 0.000 claims description 19
- 239000002157 polynucleotide Substances 0.000 claims description 19
- 102000040430 polynucleotide Human genes 0.000 claims description 19
- 239000000203 mixture Substances 0.000 claims description 18
- 238000000034 method Methods 0.000 claims description 16
- 238000001476 gene delivery Methods 0.000 claims description 15
- 102100040243 Microtubule-associated protein tau Human genes 0.000 claims description 13
- 101710115937 Microtubule-associated protein tau Proteins 0.000 claims description 12
- 101150070547 MAPT gene Proteins 0.000 claims description 11
- 210000004027 cell Anatomy 0.000 claims description 10
- 230000014509 gene expression Effects 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 6
- 101710163270 Nuclease Proteins 0.000 claims description 5
- 208000034799 Tauopathies Diseases 0.000 claims description 5
- 239000002773 nucleotide Substances 0.000 claims description 5
- 125000003729 nucleotide group Chemical group 0.000 claims description 5
- 230000004913 activation Effects 0.000 claims description 3
- 230000022532 regulation of transcription, DNA-dependent Effects 0.000 claims description 3
- 230000001105 regulatory effect Effects 0.000 claims description 3
- 230000002103 transcriptional effect Effects 0.000 claims description 3
- 230000035772 mutation Effects 0.000 claims 8
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims 2
- 239000011701 zinc Substances 0.000 claims 2
- 229910052725 zinc Inorganic materials 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 230000005764 inhibitory process Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 102000013498 tau Proteins Human genes 0.000 description 10
- 108010026424 tau Proteins Proteins 0.000 description 10
- 210000004556 brain Anatomy 0.000 description 6
- 238000002347 injection Methods 0.000 description 6
- 239000007924 injection Substances 0.000 description 6
- 239000013598 vector Substances 0.000 description 6
- 241000702421 Dependoparvovirus Species 0.000 description 5
- 210000003169 central nervous system Anatomy 0.000 description 5
- 238000007917 intracranial administration Methods 0.000 description 4
- 102000004169 proteins and genes Human genes 0.000 description 4
- 210000001320 hippocampus Anatomy 0.000 description 3
- 238000000185 intracerebroventricular administration Methods 0.000 description 3
- 238000007913 intrathecal administration Methods 0.000 description 3
- 238000001990 intravenous administration Methods 0.000 description 3
- 210000001577 neostriatum Anatomy 0.000 description 3
- 210000002569 neuron Anatomy 0.000 description 3
- 108020005004 Guide RNA Proteins 0.000 description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 description 2
- 230000002776 aggregation Effects 0.000 description 2
- 238000004220 aggregation Methods 0.000 description 2
- 210000000278 spinal cord Anatomy 0.000 description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 1
- 108700028369 Alleles Proteins 0.000 description 1
- 102000013455 Amyloid beta-Peptides Human genes 0.000 description 1
- 108010090849 Amyloid beta-Peptides Proteins 0.000 description 1
- 102000008682 Argonaute Proteins Human genes 0.000 description 1
- 108010088141 Argonaute Proteins Proteins 0.000 description 1
- 108091033409 CRISPR Proteins 0.000 description 1
- 238000010354 CRISPR gene editing Methods 0.000 description 1
- 206010018341 Gliosis Diseases 0.000 description 1
- 238000012347 Morris Water Maze Methods 0.000 description 1
- 208000016285 Movement disease Diseases 0.000 description 1
- 206010029350 Neurotoxicity Diseases 0.000 description 1
- 206010044221 Toxic encephalopathy Diseases 0.000 description 1
- 108700005077 Viral Genes Proteins 0.000 description 1
- 238000009825 accumulation Methods 0.000 description 1
- 101150010487 are gene Proteins 0.000 description 1
- 210000004208 basal nucleus of meynert Anatomy 0.000 description 1
- 239000000090 biomarker Substances 0.000 description 1
- 238000001574 biopsy Methods 0.000 description 1
- 210000001185 bone marrow Anatomy 0.000 description 1
- 210000000133 brain stem Anatomy 0.000 description 1
- 210000001638 cerebellum Anatomy 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 230000019771 cognition Effects 0.000 description 1
- 230000003920 cognitive function Effects 0.000 description 1
- 230000003750 conditioning effect Effects 0.000 description 1
- 230000008602 contraction Effects 0.000 description 1
- 230000001054 cortical effect Effects 0.000 description 1
- 230000003412 degenerative effect Effects 0.000 description 1
- 210000001353 entorhinal cortex Anatomy 0.000 description 1
- 230000003492 excitotoxic effect Effects 0.000 description 1
- 231100000063 excitotoxicity Toxicity 0.000 description 1
- 210000005153 frontal cortex Anatomy 0.000 description 1
- 238000001415 gene therapy Methods 0.000 description 1
- 230000007387 gliosis Effects 0.000 description 1
- 230000003394 haemopoietic effect Effects 0.000 description 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 1
- 230000000971 hippocampal effect Effects 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000007912 intraperitoneal administration Methods 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 210000001259 mesencephalon Anatomy 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 210000002241 neurite Anatomy 0.000 description 1
- 230000019581 neuron apoptotic process Effects 0.000 description 1
- 230000008587 neuronal excitability Effects 0.000 description 1
- 230000007135 neurotoxicity Effects 0.000 description 1
- 231100000228 neurotoxicity Toxicity 0.000 description 1
- 239000013612 plasmid Substances 0.000 description 1
- 238000011808 rodent model Methods 0.000 description 1
- 238000007920 subcutaneous administration Methods 0.000 description 1
- 210000003523 substantia nigra Anatomy 0.000 description 1
- 230000001629 suppression Effects 0.000 description 1
- 230000002459 sustained effect Effects 0.000 description 1
- 208000024891 symptom Diseases 0.000 description 1
- 230000007470 synaptic degeneration Effects 0.000 description 1
- 210000001103 thalamus Anatomy 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 230000000699 topical effect Effects 0.000 description 1
- 231100000419 toxicity Toxicity 0.000 description 1
- 230000001988 toxicity Effects 0.000 description 1
- 108700026220 vif Genes Proteins 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023053704A JP2023078446A (ja) | 2016-12-01 | 2023-03-29 | タウモジュレーターならびにその送達のための方法および組成物 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662428871P | 2016-12-01 | 2016-12-01 | |
| US62/428,871 | 2016-12-01 | ||
| US201762450895P | 2017-01-26 | 2017-01-26 | |
| US62/450,895 | 2017-01-26 | ||
| US201762466198P | 2017-03-02 | 2017-03-02 | |
| US62/466,198 | 2017-03-02 | ||
| US201762500807P | 2017-05-03 | 2017-05-03 | |
| US62/500,807 | 2017-05-03 | ||
| US201762584342P | 2017-11-10 | 2017-11-10 | |
| US62/584,342 | 2017-11-10 | ||
| PCT/US2017/064181 WO2018102665A1 (en) | 2016-12-01 | 2017-12-01 | Tau modulators and methods and compositions for delivery thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023053704A Division JP2023078446A (ja) | 2016-12-01 | 2023-03-29 | タウモジュレーターならびにその送達のための方法および組成物 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2020500526A JP2020500526A (ja) | 2020-01-16 |
| JP2020500526A5 JP2020500526A5 (https=) | 2022-08-05 |
| JPWO2018102665A5 true JPWO2018102665A5 (https=) | 2022-08-05 |
| JP7292204B2 JP7292204B2 (ja) | 2023-06-16 |
Family
ID=62239970
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019529263A Active JP7292204B2 (ja) | 2016-12-01 | 2017-12-01 | タウモジュレーターならびにその送達のための方法および組成物 |
| JP2023053704A Pending JP2023078446A (ja) | 2016-12-01 | 2023-03-29 | タウモジュレーターならびにその送達のための方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023053704A Pending JP2023078446A (ja) | 2016-12-01 | 2023-03-29 | タウモジュレーターならびにその送達のための方法および組成物 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US11504389B2 (https=) |
| EP (1) | EP3548616A4 (https=) |
| JP (2) | JP7292204B2 (https=) |
| KR (1) | KR102674612B1 (https=) |
| CN (1) | CN110214184B (https=) |
| AU (1) | AU2017367722B2 (https=) |
| CA (1) | CA3043635A1 (https=) |
| IL (1) | IL266862B2 (https=) |
| MX (1) | MX2019006426A (https=) |
| WO (1) | WO2018102665A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20210070344A (ko) * | 2018-10-02 | 2021-06-14 | 상가모 테라퓨틱스, 인코포레이티드 | 타우 단백질의 조정을 위한 방법 및 조성물 |
| CA3115158A1 (en) * | 2018-10-02 | 2020-04-09 | Sangamo Therapeutics, Inc. | Engineered genetic modulators |
| MA55625A (fr) * | 2019-04-12 | 2022-02-16 | Encoded Therapeutics Inc | Compositions et méthodes d'administration de produits thérapeutiques |
| 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. |
| KR20220071248A (ko) * | 2019-10-02 | 2022-05-31 | 상가모 테라퓨틱스, 인코포레이티드 | 알파-시누클레인 발현을 억제시키기 위한 아연 핑거 단백질 전사 인자 |
| IL293397A (en) | 2019-12-04 | 2022-07-01 | Sangamo Therapeutics Inc | New preparations and methods for the production of recombinant aav |
| AU2021209699A1 (en) * | 2020-01-22 | 2022-07-21 | Sangamo Therapeutics, Inc. | Zinc finger protein transcription factors for repressing tau expression |
| WO2022009987A1 (en) * | 2020-07-09 | 2022-01-13 | Modalis Therapeutics Corporation | Method for treating alzheimer's disease by targeting mapt gene |
| CN112640847B (zh) * | 2020-12-30 | 2022-12-13 | 重庆医科大学附属第一医院 | 一种内源性癫痫发作动物模型及其构建方法 |
| WO2025193848A1 (en) * | 2024-03-13 | 2025-09-18 | Sangamo Therapeutics, Inc. | Delivery of tau repressors using the blood-brain barrier penetrant capsid |
Family Cites Families (85)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| DE69534629D1 (de) | 1994-01-18 | 2005-12-29 | Scripps Research Inst | Derivate von zinkfingerproteinen und methoden |
| JP4285766B2 (ja) | 1994-03-23 | 2009-06-24 | オハイオ ユニバーシティ | 緻密にした核酸および細胞へのデリバリ |
| 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 |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| DE69942334D1 (de) | 1998-03-02 | 2010-06-17 | Massachusetts Inst Technology | Poly-zinkfinger-proteine mit verbesserten linkern |
| DE69929600T2 (de) | 1998-05-27 | 2006-09-07 | Avigen Inc., Alameda | Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| 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 |
| 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 |
| 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 |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| US20030044787A1 (en) | 2000-05-16 | 2003-03-06 | Joung J. Keith | Methods and compositions for interaction trap assays |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| AU2002228841C1 (en) | 2000-12-07 | 2006-11-23 | Sangamo Biosciences, Inc | Regulation of angiogenesis with zinc finger proteins |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| US7182944B2 (en) | 2001-04-25 | 2007-02-27 | The United States Of America As Represented By The Department Of Health And Human Services | Methods of increasing distribution of nucleic acids |
| US20040224385A1 (en) | 2001-08-20 | 2004-11-11 | Barbas Carlos F | Zinc finger binding domains for cnn |
| AU2002359284A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| 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 |
| EP2508608A1 (en) * | 2003-06-09 | 2012-10-10 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| 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 |
| JPWO2005030959A1 (ja) * | 2003-09-25 | 2006-12-07 | 千葉県 | 神経芽細胞腫予後診断のためのマイクロアレイと神経芽細胞腫予後診断方法 |
| US20060239966A1 (en) | 2003-10-20 | 2006-10-26 | Tornoee Jens | In vivo gene therapy of parkinson's disease |
| PT1807009E (pt) | 2004-10-05 | 2015-02-25 | Univ California | Cânula escalonada |
| 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 |
| AU2006283189B2 (en) | 2005-08-23 | 2013-01-31 | The Regents Of The University Of California | Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery |
| WO2007127803A2 (en) | 2006-04-25 | 2007-11-08 | The Regents Of The University Of California | Administration of growth factors for the treatment of cns disorders |
| 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 |
| US7837668B2 (en) | 2006-10-10 | 2010-11-23 | Ceregene, Inc. | Needle assembly for use in delivering precise dosages of proteinaceous pharmaceutical compositions and methods for use of same |
| WO2008157688A2 (en) | 2007-06-19 | 2008-12-24 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse phosphorothioate analogs of the messenger rna cap |
| CA2700231C (en) | 2007-09-27 | 2018-09-18 | Sangamo Biosciences, Inc. | Rapid in vivo identification of biologically active nucleases |
| KR101802393B1 (ko) | 2008-06-10 | 2017-11-28 | 상가모 테라퓨틱스, 인코포레이티드 | Ｂａｘ- 및 ｂａｋ-결함 세포주들의 제조 방법 및 조성물 |
| SG191561A1 (en) | 2008-08-22 | 2013-07-31 | Sangamo Biosciences Inc | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| US20110016539A1 (en) | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of neurotransmission-related genes in animals |
| US20110023153A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in alzheimer's disease |
| KR20100080068A (ko) * | 2008-12-31 | 2010-07-08 | 주식회사 툴젠 | 신규한 징크 핑거 뉴클레아제 및 이의 용도 |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| UA107571C2 (xx) | 2009-04-03 | 2015-01-26 | Фармацевтична композиція | |
| JP5866283B2 (ja) | 2009-07-28 | 2016-02-17 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 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 |
| BR112012002291A2 (pt) | 2009-07-31 | 2016-11-29 | Ethris Gmbh | "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos" |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| US20110135613A1 (en) * | 2009-12-03 | 2011-06-09 | The J. David Gladstone Institutes | Methods for treating apolipoprotein e4-associated disorders |
| EP3594333B1 (en) | 2010-02-08 | 2023-11-01 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| PT2566972T (pt) | 2010-05-03 | 2020-03-02 | Sangamo Biosciences Inc | Composições para a ligação de módulos de dedo de zinco |
| JP6208580B2 (ja) | 2010-05-17 | 2017-10-04 | サンガモ セラピューティクス， インコーポレイテッド | 新規のｄｎａ結合タンパク質及びその使用 |
| US9322023B2 (en) | 2011-10-06 | 2016-04-26 | Cornell University | Constructs and methods for the assembly of biological pathways |
| AU2012340213B2 (en) | 2011-11-16 | 2017-12-07 | Sangamo Therapeutics, Inc. | Modified DNA-binding proteins and uses thereof |
| CN108285491B (zh) | 2012-02-29 | 2021-08-10 | 桑格摩生物科学股份有限公司 | 治疗亨廷顿氏病的方法和组合物 |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| ES2807379T3 (es) * | 2013-03-14 | 2021-02-22 | Ionis Pharmaceuticals Inc | Composiciones y métodos para regular la expresión de Tau |
| CN105683376A (zh) | 2013-05-15 | 2016-06-15 | 桑格摩生物科学股份有限公司 | 用于治疗遗传病状的方法和组合物 |
| EP3004370B1 (en) | 2013-06-05 | 2024-08-21 | Duke University | Rna-guided gene editing and gene regulation |
| TWI772856B (zh) | 2013-07-19 | 2022-08-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
| US9359599B2 (en) * | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| JP2017505756A (ja) * | 2013-12-13 | 2017-02-23 | ザ ジェネラル ホスピタル コーポレイション | 可溶性高分子量（ｈｍｗ）タウ種およびその用途 |
| CN106459894B (zh) | 2014-03-18 | 2020-02-18 | 桑格摩生物科学股份有限公司 | 用于调控锌指蛋白表达的方法和组合物 |
| WO2015153760A2 (en) * | 2014-04-01 | 2015-10-08 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a nervous system disorder |
| RU2691102C2 (ru) | 2014-05-08 | 2019-06-11 | Сангамо Байосайенсиз, Инк. | Способы и композиции для лечения болезни хантингтона |
| EP3152319A4 (en) * | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
| AU2016215155A1 (en) * | 2015-02-04 | 2017-08-17 | F. Hoffmann-La Roche Ag | Tau antisense oligomers and uses thereof |
| US20170035860A1 (en) * | 2015-04-02 | 2017-02-09 | Alexander C. Flynn | Compositions and methods for treatment of neurogenerative diseases |
| WO2017011556A1 (en) | 2015-07-13 | 2017-01-19 | The General Hospital Corporation | Rare phosphorylated high molecular weight (hmw) tau species that are involved in neuronal uptake and propagation and applications thereof |
| CA3002524A1 (en) | 2015-10-28 | 2017-05-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| WO2017197141A2 (en) | 2016-05-13 | 2017-11-16 | Sangamo Therapeutics, Inc. | Targeted treatment of androgenic alopecia |
| SG10201913948PA (en) | 2016-08-24 | 2020-03-30 | Sangamo Therapeutics Inc | Engineered target specific nucleases |
| LT3504229T (lt) * | 2016-08-24 | 2021-12-10 | Sangamo Therapeutics, Inc. | Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes |
| WO2018039471A2 (en) | 2016-08-25 | 2018-03-01 | Trustees Of Boston University | Synthetic transcriptional and epigenetic regulators based on engineered, orthogonal zinc finger proteins |
| WO2018049009A2 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| US11371023B2 (en) | 2016-11-22 | 2022-06-28 | Wisconsin Alumni Research Foundation | Artificial transcription factors and uses thereof |
| KR20210070344A (ko) | 2018-10-02 | 2021-06-14 | 상가모 테라퓨틱스, 인코포레이티드 | 타우 단백질의 조정을 위한 방법 및 조성물 |
| CA3115158A1 (en) | 2018-10-02 | 2020-04-09 | Sangamo Therapeutics, Inc. | Engineered genetic modulators |
| AU2021209699A1 (en) | 2020-01-22 | 2022-07-21 | Sangamo Therapeutics, Inc. | Zinc finger protein transcription factors for repressing tau expression |
-
2017
- 2017-12-01 CN CN201780084345.1A patent/CN110214184B/zh active Active
- 2017-12-01 WO PCT/US2017/064181 patent/WO2018102665A1/en not_active Ceased
- 2017-12-01 MX MX2019006426A patent/MX2019006426A/es unknown
- 2017-12-01 EP EP17876620.0A patent/EP3548616A4/en active Pending
- 2017-12-01 CA CA3043635A patent/CA3043635A1/en active Pending
- 2017-12-01 JP JP2019529263A patent/JP7292204B2/ja active Active
- 2017-12-01 KR KR1020197016870A patent/KR102674612B1/ko active Active
- 2017-12-01 IL IL266862A patent/IL266862B2/en unknown
- 2017-12-01 AU AU2017367722A patent/AU2017367722B2/en active Active
- 2017-12-01 US US15/828,931 patent/US11504389B2/en active Active
-
2022
- 2022-10-13 US US18/046,327 patent/US20230270774A1/en active Pending
-
2023
- 2023-03-29 JP JP2023053704A patent/JP2023078446A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7436089B2 (ja) | 前頭側頭型認知症の治療 | |
| US20250161485A1 (en) | Aav capsid variants and uses thereof | |
| KR102527259B1 (ko) | 스터퍼/필러 폴리누클레오티드 서열을 포함하는 벡터 및 사용 방법 | |
| JP2020533959A5 (https=) | ||
| Bowers et al. | Genetic therapy for the nervous system | |
| KR20250025387A (ko) | Aav 캡시드 변이체 및 이의 용도 | |
| Miyake et al. | Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors | |
| JP7616995B2 (ja) | 神経変性疾患のための遺伝子治療 | |
| Cappa et al. | Gene therapy for rare central nervous system diseases comes to age | |
| JP2021525245A5 (https=) | ||
| JP2022531809A (ja) | ＡＴＰａｓｅ媒介性疾患の処置のための組成物および方法 | |
| JPWO2018102665A5 (https=) | ||
| TW202417467A (zh) | Ａａｖ蛋白殼變異體及其用途 | |
| WO2025038430A1 (en) | Aav capsid variants and uses thereof | |
| TW202424201A (zh) | Ａａｖ殼體變異體及其用途 | |
| WO2025147436A1 (en) | Aav capsid variants and uses thereof | |
| JPWO2019226832A5 (https=) | ||
| TWI883636B (zh) | 含有星狀細胞導向啟動子構築體之合成核酸及其使用方法 | |
| JPWO2021028299A5 (https=) | ||
| Miyake et al. | Gene delivery into the central nervous system (CNS) using AAV vectors | |
| US20240100132A1 (en) | Modulation of aav-based gene expression | |
| US20260103728A1 (en) | Method of treating lbsl by enhancing dars2 expression | |
| CN111093716A (zh) | 用于治疗神经性疼痛的方法和组合物 | |
| WO2024259064A1 (en) | Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom | |
| WO2024154102A1 (en) | Compositions and methods for administration of therapeutic and diagnostic agents to the central nervous system |