MX2021014868A - Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas. - Google Patents
Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas.Info
- Publication number
- MX2021014868A MX2021014868A MX2021014868A MX2021014868A MX2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- oligonucleotides
- neurological diseases
- treatment
- diseases
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- 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
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Steroid Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962856264P | 2019-06-03 | 2019-06-03 | |
| US201962914252P | 2019-10-11 | 2019-10-11 | |
| US201962949817P | 2019-12-18 | 2019-12-18 | |
| PCT/US2020/035811 WO2020247419A2 (en) | 2019-06-03 | 2020-06-03 | Oligonucleotides and methods of use for treating neurological diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021014868A true MX2021014868A (es) | 2022-03-25 |
Family
ID=73653367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021014868A MX2021014868A (es) | 2019-06-03 | 2020-06-03 | Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220333105A1 (OSRAM) |
| EP (1) | EP3976010A4 (OSRAM) |
| JP (2) | JP7795914B2 (OSRAM) |
| KR (1) | KR20220033472A (OSRAM) |
| CN (1) | CN114555069A (OSRAM) |
| AU (1) | AU2020288555A1 (OSRAM) |
| BR (1) | BR112021024463A2 (OSRAM) |
| CA (1) | CA3142526A1 (OSRAM) |
| IL (1) | IL288574A (OSRAM) |
| MX (1) | MX2021014868A (OSRAM) |
| PH (1) | PH12021553024A1 (OSRAM) |
| WO (1) | WO2020247419A2 (OSRAM) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN114173821A (zh) * | 2019-01-14 | 2022-03-11 | 哈佛学院校长同事会 | 用于恢复stmn2水平的方法和组合物 |
| EP4127172A4 (en) * | 2020-03-25 | 2025-06-04 | President and Fellows of Harvard College | Methods and compositions for restoring STMN2 levels |
| KR20230043819A (ko) * | 2020-06-03 | 2023-03-31 | 큐랄리스 코포레이션 | 유전자 전사체의 조정제를 사용한 신경계 질환의 치료 |
| WO2022018155A1 (en) * | 2020-07-23 | 2022-01-27 | F. Hoffmann-La Roche Ag | Lna oligonucleotides for splice modulation of stmn2 |
| WO2022018187A1 (en) * | 2020-07-23 | 2022-01-27 | F. Hoffmann-La Roche Ag | Oligonucleotides targeting rna binding protein sites |
| AU2022255175A1 (en) * | 2021-04-06 | 2023-11-23 | Trace Neuroscience, Inc. | Compositions and methods for treating tdp-43 proteinopathy |
| IL310616A (en) * | 2021-08-11 | 2024-04-01 | Arbor Biotechnologies Inc | Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof |
| CA3231484A1 (en) | 2021-10-01 | 2023-04-06 | Qinwen Mao | Pathologic tdp-43 as a biomarker for the diagnosis of tdp-43 proteinopathy |
| AU2022402929A1 (en) * | 2021-12-03 | 2024-06-13 | Quralis Corporation | Splice switcher antisense oligonucleotides with modified backbone chemistries |
| GB202208387D0 (en) * | 2022-06-08 | 2022-07-20 | Ucl Business Ltd | Modified U7 snRNA construct |
| GB202208384D0 (en) * | 2022-06-08 | 2022-07-20 | Ucl Business Ltd | Modified U7 snRNA construct |
| EP4630558A2 (en) * | 2022-12-09 | 2025-10-15 | Auttx, LLC | Targeted treatment of spliceopathy-induced neurological disorders |
| CN120712355A (zh) * | 2023-02-15 | 2025-09-26 | 阿伯生物技术公司 | 用于抑制抑微管装配蛋白2（stmn2）转录物中的异常剪接的基因编辑方法 |
| WO2025184347A1 (en) * | 2024-02-27 | 2025-09-04 | Emory University | Network analysis of the cerebrospinal fluid proteome of sporadic and familial forms of amyotrophic lateral sclerosis and methods of treatment of the same |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6187545B1 (en) * | 2000-01-19 | 2001-02-13 | Isis Pharmaceuticals Inc. | Antisense modulation of pepck-cytosolic expression |
| WO2002034908A2 (en) * | 2000-10-23 | 2002-05-02 | Mermaid Pharmaceuticals Gmbh | Method for temporally controlling antisense-mediated gene inactivation |
| US20030104410A1 (en) * | 2001-03-16 | 2003-06-05 | Affymetrix, Inc. | Human microarray |
| JP2007104971A (ja) | 2005-10-14 | 2007-04-26 | New Industry Research Organization | ミオスタチンのスプライシング制御アンチセンスオリゴヌクレオチド |
| EP1815863A1 (en) * | 2006-02-03 | 2007-08-08 | Nederlandse Organisatie voor toegepast-natuurwetenschappelijk Onderzoek TNO | Use of TLR3 agonists for the treatment of neurodegenerative disorders |
| WO2008112903A2 (en) * | 2007-03-13 | 2008-09-18 | The Children's Hospital Of Philadelphia | Genetic alterations on chromosome 16 and methods of use thereof for the diagnosis and treatment of type 1 diabetes |
| CN102549158B (zh) * | 2009-05-22 | 2017-09-26 | 库尔纳公司 | 通过抑制针对转录因子e3(tfe3)的天然反义转录物来治疗tfe3和胰岛素受体底物蛋白2(irs2)相关的疾病 |
| WO2012005898A2 (en) * | 2010-06-15 | 2012-01-12 | Alnylam Pharmaceuticals, Inc. | Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof |
| WO2012177639A2 (en) * | 2011-06-22 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Bioprocessing and bioproduction using avian cell lines |
| WO2013086441A2 (en) | 2011-12-08 | 2013-06-13 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| CN111254145A (zh) | 2013-03-14 | 2020-06-09 | Ionis制药公司 | 用于调节tau表达的组合物和方法 |
| WO2015051239A1 (en) * | 2013-10-03 | 2015-04-09 | The General Hospital Corporation | Methods for increasing neuronal survival |
| US10538762B2 (en) * | 2014-10-14 | 2020-01-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat |
| JP2018537528A (ja) * | 2015-11-04 | 2018-12-20 | アイデラ・ファーマシューティカルズ，インコーポレーテッド | Ｎｌｒｐ３遺伝子発現を阻害するための組成物およびその使用 |
| US11833168B2 (en) | 2018-06-14 | 2023-12-05 | Ionis Pharmaceuticals, Inc. | Compounds and methods for increasing STMN2 expression |
| EP4127172A4 (en) * | 2020-03-25 | 2025-06-04 | President and Fellows of Harvard College | Methods and compositions for restoring STMN2 levels |
-
2020
- 2020-06-03 JP JP2021571911A patent/JP7795914B2/ja active Active
- 2020-06-03 EP EP20818770.8A patent/EP3976010A4/en active Pending
- 2020-06-03 AU AU2020288555A patent/AU2020288555A1/en active Pending
- 2020-06-03 KR KR1020217043411A patent/KR20220033472A/ko active Pending
- 2020-06-03 BR BR112021024463A patent/BR112021024463A2/pt unknown
- 2020-06-03 US US17/616,350 patent/US20220333105A1/en active Pending
- 2020-06-03 CN CN202080054507.9A patent/CN114555069A/zh active Pending
- 2020-06-03 MX MX2021014868A patent/MX2021014868A/es unknown
- 2020-06-03 PH PH1/2021/553024A patent/PH12021553024A1/en unknown
- 2020-06-03 CA CA3142526A patent/CA3142526A1/en active Pending
- 2020-06-03 WO PCT/US2020/035811 patent/WO2020247419A2/en not_active Ceased
-
2021
- 2021-12-01 IL IL288574A patent/IL288574A/en unknown
-
2025
- 2025-08-08 JP JP2025133172A patent/JP2026002847A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP7795914B2 (ja) | 2026-01-08 |
| JP2022536085A (ja) | 2022-08-12 |
| WO2020247419A3 (en) | 2021-01-14 |
| AU2020288555A1 (en) | 2022-01-20 |
| KR20220033472A (ko) | 2022-03-16 |
| EP3976010A2 (en) | 2022-04-06 |
| JP2026002847A (ja) | 2026-01-08 |
| PH12021553024A1 (en) | 2023-08-23 |
| CA3142526A1 (en) | 2020-12-10 |
| EP3976010A4 (en) | 2023-08-02 |
| BR112021024463A2 (pt) | 2022-03-08 |
| WO2020247419A2 (en) | 2020-12-10 |
| IL288574A (en) | 2022-02-01 |
| US20220333105A1 (en) | 2022-10-20 |
| CN114555069A (zh) | 2022-05-27 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| BR112021024463A2 (pt) | Oligonucleotídeos e métodos de uso para o tratamento de doenças neurológicas | |
| MX2021010193A (es) | Azepino-indoles y otros heterociclos para el tratamiento de trastornos cerebrales. | |
| CO2019003846A2 (es) | Tratamiento aav de la enfermedad de huntington | |
| CL2021002585A1 (es) | Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central | |
| CO2019010355A2 (es) | Arn terapéutico | |
| CL2019002427A1 (es) | Inhibición de smarca2 para el tratamiento de cáncer. | |
| NZ733882A (en) | Compositions for modulating c9orf72 expression | |
| SV2018005614A (es) | Inhibidores peptidicos del receptor de interleucina 23 y uso para tratar enfermedades inflamatorias | |
| EA201892431A1 (ru) | Олигонуклеотиды для лечения заболевания глаз | |
| MX2018005315A (es) | Composiciones y metodos para el tratamiento del cancer. | |
| PH12017500601A1 (en) | Anti-tnf compounds | |
| MX391815B (es) | AGENTES DE iARN, COMPOSICIONES Y METODOS DE LOS MISMOS PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES ASOCIADAS CON TRANSTITERINA (TTR). | |
| PE20181448A1 (es) | Composiciones y metodos para disminuir la expresion de tau | |
| MX2021014206A (es) | Oligonucleotidos gapmer modificados y metodos de uso. | |
| MX386146B (es) | Métodos para tratar esclerosis múltiple usando compuestos de pirimidina y piridina con actividad inhibidora de tirosina cinasa de bruton. | |
| IL269735A (en) | Antisense oligonucleotides for the treatment of stargardt disease | |
| ECSP20077106A (es) | Régimen de dosificación para el tratamiento de trastornos relacionados con pi3k | |
| AR104755A1 (es) | Método para el tratamiento de enfermedad neurológica | |
| CY1123639T1 (el) | Μονοκλωνα ολιγονουκλεοτιδια για χρηση στην ιατρικη θεραπευτικη αντιμετωπιση διαταραχων του δερματος | |
| EP3384028A4 (en) | MONOCARBOXYLATE TRANSPORTER 4 (MCT4) ANTISENSE OLIGONUCLEOTIDE (ASO) INHIBITORS FOR USE AS THERAPEUTICS IN THE TREATMENT OF CANCER | |
| EA201791986A1 (ru) | Олигонуклеотиды, соответствующие экзону 73 гена col7a1, для терапии буллезного эпидермолиза | |
| MX388737B (es) | Oligonucleótidos terapéuticos. | |
| UY38352A (es) | Inhibidores de integrina alfavbeta6 | |
| CL2020002038A1 (es) | Oligonucleótidos para modular la expresión de tmem106b. | |
| EA201792406A1 (ru) | Антисмысловые олигонуклеотиды для лечения дистрофического буллезного эпидермолиза |