ES2582649T3 - Tratamiento de afecciones del SNC - Google Patents
Tratamiento de afecciones del SNC Download PDFInfo
- Publication number
- ES2582649T3 ES2582649T3 ES07733553.7T ES07733553T ES2582649T3 ES 2582649 T3 ES2582649 T3 ES 2582649T3 ES 07733553 T ES07733553 T ES 07733553T ES 2582649 T3 ES2582649 T3 ES 2582649T3
- Authority
- ES
- Spain
- Prior art keywords
- sirna
- treatment
- seq
- cns
- sequence selected
- 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.)
- Active
Links
- 108020004459 Small interfering RNA Proteins 0.000 abstract description 8
- 238000002360 preparation method Methods 0.000 abstract description 3
- 230000014509 gene expression Effects 0.000 abstract description 2
- 210000003169 central nervous system Anatomy 0.000 abstract 2
- 239000003814 drug Substances 0.000 abstract 2
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 abstract 1
- 230000002159 abnormal effect Effects 0.000 abstract 1
- 208000015114 central nervous system disease Diseases 0.000 abstract 1
- 229940079593 drug Drugs 0.000 abstract 1
- 230000009368 gene silencing by RNA Effects 0.000 abstract 1
- 230000000626 neurodegenerative effect Effects 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 108010048367 enhanced green fluorescent protein Proteins 0.000 description 7
- 241000699670 Mus sp. Species 0.000 description 6
- 210000004027 cell Anatomy 0.000 description 4
- 108020004999 messenger RNA Proteins 0.000 description 4
- 239000000725 suspension Substances 0.000 description 4
- 239000013598 vector Substances 0.000 description 4
- 241000701022 Cytomegalovirus Species 0.000 description 3
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 description 3
- 241001465754 Metazoa Species 0.000 description 3
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 3
- 239000002299 complementary DNA Substances 0.000 description 3
- 239000003623 enhancer Substances 0.000 description 3
- 150000002148 esters Chemical class 0.000 description 3
- 239000002904 solvent Substances 0.000 description 3
- 238000011830 transgenic mouse model Methods 0.000 description 3
- 239000003981 vehicle Substances 0.000 description 3
- PUPZLCDOIYMWBV-UHFFFAOYSA-N (+/-)-1,3-Butanediol Chemical compound CC(O)CCO PUPZLCDOIYMWBV-UHFFFAOYSA-N 0.000 description 2
- 241000283690 Bos taurus Species 0.000 description 2
- 101100268670 Caenorhabditis elegans acc-3 gene Proteins 0.000 description 2
- 150000001875 compounds Chemical class 0.000 description 2
- 235000014113 dietary fatty acids Nutrition 0.000 description 2
- 239000002270 dispersing agent Substances 0.000 description 2
- 239000000839 emulsion Substances 0.000 description 2
- 239000000194 fatty acid Substances 0.000 description 2
- 229930195729 fatty acid Natural products 0.000 description 2
- 150000004665 fatty acids Chemical class 0.000 description 2
- 235000003599 food sweetener Nutrition 0.000 description 2
- 102000018146 globin Human genes 0.000 description 2
- 108060003196 globin Proteins 0.000 description 2
- 239000005090 green fluorescent protein Substances 0.000 description 2
- 239000000346 nonvolatile oil Substances 0.000 description 2
- 108020004707 nucleic acids Proteins 0.000 description 2
- 102000039446 nucleic acids Human genes 0.000 description 2
- 150000007523 nucleic acids Chemical class 0.000 description 2
- 230000008488 polyadenylation Effects 0.000 description 2
- 239000000375 suspending agent Substances 0.000 description 2
- 239000003765 sweetening agent Substances 0.000 description 2
- 230000001225 therapeutic effect Effects 0.000 description 2
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 2
- 239000000080 wetting agent Substances 0.000 description 2
- WRIDQFICGBMAFQ-UHFFFAOYSA-N (E)-8-Octadecenoic acid Natural products CCCCCCCCCC=CCCCCCCC(O)=O WRIDQFICGBMAFQ-UHFFFAOYSA-N 0.000 description 1
- ZORQXIQZAOLNGE-UHFFFAOYSA-N 1,1-difluorocyclohexane Chemical compound FC1(F)CCCCC1 ZORQXIQZAOLNGE-UHFFFAOYSA-N 0.000 description 1
- IIZPXYDJLKNOIY-JXPKJXOSSA-N 1-palmitoyl-2-arachidonoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCC\C=C/C\C=C/C\C=C/C\C=C/CCCCC IIZPXYDJLKNOIY-JXPKJXOSSA-N 0.000 description 1
- LQJBNNIYVWPHFW-UHFFFAOYSA-N 20:1omega9c fatty acid Natural products CCCCCCCCCCC=CCCCCCCCC(O)=O LQJBNNIYVWPHFW-UHFFFAOYSA-N 0.000 description 1
- QSBYPNXLFMSGKH-UHFFFAOYSA-N 9-Heptadecensaeure Natural products CCCCCCCC=CCCCCCCCC(O)=O QSBYPNXLFMSGKH-UHFFFAOYSA-N 0.000 description 1
- 102000007469 Actins Human genes 0.000 description 1
- 108010085238 Actins Proteins 0.000 description 1
- 241000416162 Astragalus gummifer Species 0.000 description 1
- 108091026890 Coding region Proteins 0.000 description 1
- IAYPIBMASNFSPL-UHFFFAOYSA-N Ethylene oxide Chemical compound C1CO1 IAYPIBMASNFSPL-UHFFFAOYSA-N 0.000 description 1
- 229920000084 Gum arabic Polymers 0.000 description 1
- 241000699660 Mus musculus Species 0.000 description 1
- 239000005642 Oleic acid Substances 0.000 description 1
- ZQPPMHVWECSIRJ-UHFFFAOYSA-N Oleic acid Natural products CCCCCCCCC=CCCCCCCCC(O)=O ZQPPMHVWECSIRJ-UHFFFAOYSA-N 0.000 description 1
- 241000978776 Senegalia senegal Species 0.000 description 1
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical class O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 description 1
- 229920001615 Tragacanth Polymers 0.000 description 1
- 108700019146 Transgenes Proteins 0.000 description 1
- 235000010489 acacia gum Nutrition 0.000 description 1
- 239000000205 acacia gum Substances 0.000 description 1
- 150000008064 anhydrides Chemical class 0.000 description 1
- 230000000692 anti-sense effect Effects 0.000 description 1
- 230000009286 beneficial effect Effects 0.000 description 1
- 239000003086 colorant Substances 0.000 description 1
- 239000007859 condensation product Substances 0.000 description 1
- 238000010276 construction Methods 0.000 description 1
- 239000003085 diluting agent Substances 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 239000003995 emulsifying agent Substances 0.000 description 1
- 210000003743 erythrocyte Anatomy 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- 239000000796 flavoring agent Substances 0.000 description 1
- 235000013355 food flavoring agent Nutrition 0.000 description 1
- FBPFZTCFMRRESA-UHFFFAOYSA-N hexane-1,2,3,4,5,6-hexol Chemical compound OCC(O)C(O)C(O)C(O)CO FBPFZTCFMRRESA-UHFFFAOYSA-N 0.000 description 1
- 229940102223 injectable solution Drugs 0.000 description 1
- 229940102213 injectable suspension Drugs 0.000 description 1
- 238000007918 intramuscular administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- QXJSBBXBKPUZAA-UHFFFAOYSA-N isooleic acid Natural products CCCCCCCC=CCCCCCCCCC(O)=O QXJSBBXBKPUZAA-UHFFFAOYSA-N 0.000 description 1
- 235000010445 lecithin Nutrition 0.000 description 1
- 239000000787 lecithin Substances 0.000 description 1
- 229940067606 lecithin Drugs 0.000 description 1
- 150000002632 lipids Chemical class 0.000 description 1
- 238000000034 method Methods 0.000 description 1
- 239000002480 mineral oil Substances 0.000 description 1
- 235000010446 mineral oil Nutrition 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 231100000252 nontoxic Toxicity 0.000 description 1
- 230000003000 nontoxic effect Effects 0.000 description 1
- 239000003921 oil Substances 0.000 description 1
- 235000019198 oils Nutrition 0.000 description 1
- ZQPPMHVWECSIRJ-KTKRTIGZSA-N oleic acid Chemical compound CCCCCCCC\C=C/CCCCCCCC(O)=O ZQPPMHVWECSIRJ-KTKRTIGZSA-N 0.000 description 1
- 239000008194 pharmaceutical composition Substances 0.000 description 1
- 239000000546 pharmaceutical excipient Substances 0.000 description 1
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 description 1
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 description 1
- 229920000053 polysorbate 80 Polymers 0.000 description 1
- 239000003755 preservative agent Substances 0.000 description 1
- 239000001593 sorbitan monooleate Substances 0.000 description 1
- 235000011069 sorbitan monooleate Nutrition 0.000 description 1
- 229940035049 sorbitan monooleate Drugs 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 238000001890 transfection Methods 0.000 description 1
- 230000010474 transient expression Effects 0.000 description 1
- 235000015112 vegetable and seed oil Nutrition 0.000 description 1
- 239000008158 vegetable oil Substances 0.000 description 1
Classifications
-
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—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
- 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
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
- A61K2039/541—Mucosal route
- A61K2039/543—Mucosal route intranasal
-
- 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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GB0605337 | 2006-03-17 | ||
| GBGB0605337.5A GB0605337D0 (en) | 2006-03-17 | 2006-03-17 | Treatment of CNS conditions |
| PCT/GB2007/050128 WO2007107789A2 (en) | 2006-03-17 | 2007-03-16 | Treatment of cns conditions |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2582649T3 true ES2582649T3 (es) | 2016-09-14 |
Family
ID=36292900
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES07733553.7T Active ES2582649T3 (es) | 2006-03-17 | 2007-03-16 | Tratamiento de afecciones del SNC |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US8871729B2 (https=) |
| EP (1) | EP2004823B1 (https=) |
| JP (2) | JP5781722B2 (https=) |
| KR (2) | KR101669944B1 (https=) |
| CN (1) | CN101448944B (https=) |
| AU (1) | AU2007228570B2 (https=) |
| CA (1) | CA2645120C (https=) |
| ES (1) | ES2582649T3 (https=) |
| GB (1) | GB0605337D0 (https=) |
| IL (1) | IL194114A (https=) |
| MX (1) | MX2008011731A (https=) |
| RU (1) | RU2426544C2 (https=) |
| WO (1) | WO2007107789A2 (https=) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008124066A1 (en) * | 2007-04-05 | 2008-10-16 | The J. David Gladstone Institutes | Agents that reduce neuronal overexcitation |
| WO2010080452A2 (en) | 2008-12-18 | 2010-07-15 | Quark Pharmaceuticals, Inc. | siRNA COMPOUNDS AND METHODS OF USE THEREOF |
| EP3626823A1 (en) * | 2009-09-11 | 2020-03-25 | Ionis Pharmaceuticals, Inc. | Modulation of huntingtin expression |
| DK2504435T3 (da) | 2009-11-26 | 2019-12-09 | Quark Pharmaceuticals Inc | Sirna-forbindelser omfattende terminale substitutioner |
| US20110135613A1 (en) | 2009-12-03 | 2011-06-09 | The J. David Gladstone Institutes | Methods for treating apolipoprotein e4-associated disorders |
| EP2510098B1 (en) | 2009-12-09 | 2015-02-11 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the cns |
| AU2011219029B2 (en) * | 2010-02-26 | 2017-02-02 | Columbia University | Methods and compositions for the detection and treatment of cancer involving miRNAs and miRNA inhibitors and targets |
| RU2664452C2 (ru) | 2010-04-19 | 2018-08-17 | Нлифе Терапеутикс, С.Л. | Конъюгат, медицинское средство и способы лечения и/или профилактики депрессии и болезни, связанной с отложением телец Леви |
| EP2380595A1 (en) | 2010-04-19 | 2011-10-26 | Nlife Therapeutics S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| WO2012058210A1 (en) | 2010-10-29 | 2012-05-03 | Merck Sharp & Dohme Corp. | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA) |
| AU2011338682B2 (en) | 2010-12-06 | 2017-04-27 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising threose modifications |
| WO2012123819A1 (en) * | 2011-03-15 | 2012-09-20 | Optinose As Et Al | Nasal delivery |
| CA2851761C (en) * | 2011-10-14 | 2021-06-15 | Centre National De La Recherche Scientifique | Method of diagnosis, prognostic or treatment of neurodegenerative diseases |
| EP2822600A4 (en) * | 2012-03-09 | 2016-04-06 | Univ Northeastern | METHOD OF DISTRIBUTING NUCLEIC ACID ANOPARTICLES TO THE CENTRAL NERVOUS SYSTEM FOR THE TREATMENT OF DISEASES OF THE CENTRAL NERVOUS SYSTEM |
| AU2013203395A1 (en) * | 2012-03-30 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome |
| US9611473B2 (en) | 2012-09-12 | 2017-04-04 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| AU2013336581A1 (en) | 2012-10-26 | 2015-06-11 | Nlife Therapeutics, S.L. | Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
| ES2807379T3 (es) * | 2013-03-14 | 2021-02-22 | Ionis Pharmaceuticals Inc | Composiciones y métodos para regular la expresión de Tau |
| JP2016523980A (ja) * | 2013-07-11 | 2016-08-12 | ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク | タウ発現を抑制するマイクロｒｎａ |
| 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 |
| EP3027223A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| 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 |
| EP3274456B1 (en) * | 2015-03-25 | 2020-06-24 | Università Degli Studi Di Trento | Rna interference mediated therapy for neurodegenerative diseases |
| PH12018501207B1 (en) | 2015-12-21 | 2024-02-23 | Novartis Ag | Compositions and methods for decreasing tau expression |
| ES2963428T3 (es) | 2016-09-29 | 2024-03-27 | Biogen Ma Inc | Compuestos y métodos para reducir la expresión de Tau |
| BR112020006671A2 (pt) * | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| KR20200131263A (ko) | 2018-03-13 | 2020-11-23 | 얀센 파마슈티카 엔.브이. | 변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법 |
| AU2021238319A1 (en) * | 2020-03-18 | 2022-10-06 | University Of Massachusetts | Oligonucleotides for MAPT modulation |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| CN113817728A (zh) * | 2020-06-19 | 2021-12-21 | 四川大学华西医院 | 一种有效干扰Tau蛋白表达的重组慢病毒及其应用 |
| KR20240067943A (ko) * | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| CN119173631A (zh) * | 2022-05-12 | 2024-12-20 | 迪克纳制药公司 | 用于抑制mapt表达的组合物和方法 |
| KR20250129743A (ko) * | 2022-12-29 | 2025-08-29 | 보이저 테라퓨틱스, 인크. | Mapt를 조절하기 위한 조성물 및 방법 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2116460A1 (en) | 1993-03-02 | 1994-09-03 | Mitsubishi Chemical Corporation | Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being |
| US6410046B1 (en) * | 1996-11-19 | 2002-06-25 | Intrabrain International Nv | Administering pharmaceuticals to the mammalian central nervous system |
| WO2002086105A1 (en) | 2001-04-20 | 2002-10-31 | Chiron Corporation | Delivery of polynucleotide agents to the central nervous sysstem |
| US20050159376A1 (en) | 2002-02-20 | 2005-07-21 | Slrna Therapeutics, Inc. | RNA interference mediated inhibition 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA) |
| IL143379A (en) | 2001-05-24 | 2013-11-28 | Yissum Res Dev Co | Oligonucleotide against human ache isoform r and its uses |
| AU2003207708A1 (en) | 2002-02-20 | 2003-09-09 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of map kinase genes |
| GB2406568B (en) | 2002-02-20 | 2005-09-28 | Sirna Therapeutics Inc | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| CA2482904A1 (en) | 2002-04-18 | 2003-10-23 | Lynkeus Biotech Gmbh | Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification |
| US20040254146A1 (en) | 2002-05-21 | 2004-12-16 | Nastech Pharmaceutical Company Inc. | Carboxylate salts of galantamine and their pharmaceutical use |
| US20030225031A1 (en) | 2002-05-21 | 2003-12-04 | Quay Steven C. | Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20050106731A1 (en) * | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| US20040241854A1 (en) | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
| CA2500224C (en) | 2002-09-25 | 2015-04-28 | University Of Massachusetts | In vivo gene silencing by chemically modified and stable sirna |
| MXPA05003287A (es) | 2002-09-28 | 2005-07-05 | Massachusetts Inst Technology | Influenza terapeutica. |
| ES2334125T3 (es) * | 2002-11-04 | 2010-03-05 | University Of Massachusetts | Interferencia de arn especifico de alelos. |
| US7829694B2 (en) | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| EP2508608A1 (en) * | 2003-06-09 | 2012-10-10 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
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|---|---|
| AU2007228570A8 (en) | 2008-10-23 |
| HK1126812A1 (zh) | 2009-09-11 |
| KR20140076634A (ko) | 2014-06-20 |
| KR20080111063A (ko) | 2008-12-22 |
| IL194114A (en) | 2012-06-28 |
| RU2426544C2 (ru) | 2011-08-20 |
| WO2007107789A2 (en) | 2007-09-27 |
| RU2008141162A (ru) | 2010-04-27 |
| JP5781722B2 (ja) | 2015-09-24 |
| AU2007228570B2 (en) | 2013-09-19 |
| US8871729B2 (en) | 2014-10-28 |
| JP2009530257A (ja) | 2009-08-27 |
| KR101605144B1 (ko) | 2016-03-21 |
| JP5923466B2 (ja) | 2016-05-24 |
| JP2013234197A (ja) | 2013-11-21 |
| CN101448944A (zh) | 2009-06-03 |
| KR101669944B1 (ko) | 2016-10-28 |
| GB0605337D0 (en) | 2006-04-26 |
| CA2645120C (en) | 2015-02-24 |
| AU2007228570A1 (en) | 2007-09-27 |
| US20090176728A1 (en) | 2009-07-09 |
| CA2645120A1 (en) | 2007-09-27 |
| WO2007107789A3 (en) | 2008-05-08 |
| EP2004823A2 (en) | 2008-12-24 |
| CN101448944B (zh) | 2014-02-12 |
| MX2008011731A (es) | 2009-03-05 |
| IL194114A0 (en) | 2011-08-01 |
| EP2004823B1 (en) | 2016-05-25 |
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