KR20230085222A - Sod-1 발현을 조절하기 위한 조성물 - Google Patents
Sod-1 발현을 조절하기 위한 조성물 Download PDFInfo
- Publication number
- KR20230085222A KR20230085222A KR1020237018802A KR20237018802A KR20230085222A KR 20230085222 A KR20230085222 A KR 20230085222A KR 1020237018802 A KR1020237018802 A KR 1020237018802A KR 20237018802 A KR20237018802 A KR 20237018802A KR 20230085222 A KR20230085222 A KR 20230085222A
- Authority
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- South Korea
- Prior art keywords
- sod
- isis
- modified
- certain embodiments
- sugar
- 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.)
- Ceased
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- C—CHEMISTRY; METALLURGY
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- 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
- C12N15/1137—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 against enzymes
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- 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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- 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
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- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- 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
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- 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
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- 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
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- 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
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- 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
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- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- 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
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- 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 ===---===
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| KR1020247022551A KR20240111010A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461973803P | 2014-04-01 | 2014-04-01 | |
| US61/973,803 | 2014-04-01 | ||
| KR1020227017808A KR20220077933A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| PCT/US2015/023934 WO2015153800A2 (en) | 2014-04-01 | 2015-04-01 | Compositions for modulating sod-1 expression |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227017808A Division KR20220077933A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247022551A Division KR20240111010A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20230085222A true KR20230085222A (ko) | 2023-06-13 |
Family
ID=54241435
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020257023289A Pending KR20250113519A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020167030062A Active KR102285629B1 (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020237018802A Ceased KR20230085222A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020227017808A Ceased KR20220077933A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020247022551A Ceased KR20240111010A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020217024143A Ceased KR20210097225A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020257023289A Pending KR20250113519A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020167030062A Active KR102285629B1 (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020227017808A Ceased KR20220077933A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020247022551A Ceased KR20240111010A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
| KR1020217024143A Ceased KR20210097225A (ko) | 2014-04-01 | 2015-04-01 | Sod-1 발현을 조절하기 위한 조성물 |
Country Status (30)
| Country | Link |
|---|---|
| US (3) | US10385341B2 (enExample) |
| EP (3) | EP4137573A3 (enExample) |
| JP (5) | JP6622214B2 (enExample) |
| KR (6) | KR20250113519A (enExample) |
| CN (2) | CN111440798A (enExample) |
| AU (4) | AU2015240761B2 (enExample) |
| BR (1) | BR112016021848B1 (enExample) |
| CA (1) | CA2942394A1 (enExample) |
| CL (3) | CL2016002509A1 (enExample) |
| CY (1) | CY1123288T1 (enExample) |
| DK (2) | DK3757214T3 (enExample) |
| ES (2) | ES2926869T3 (enExample) |
| FI (1) | FIC20240035I1 (enExample) |
| FR (1) | FR24C1039I2 (enExample) |
| HR (2) | HRP20201078T1 (enExample) |
| HU (3) | HUE050704T2 (enExample) |
| IL (2) | IL247714B (enExample) |
| LT (3) | LT3757214T (enExample) |
| MX (2) | MX373908B (enExample) |
| MY (2) | MY192634A (enExample) |
| NL (1) | NL301293I2 (enExample) |
| NZ (1) | NZ724100A (enExample) |
| PL (2) | PL3757214T3 (enExample) |
| PT (2) | PT3126499T (enExample) |
| RS (2) | RS63487B1 (enExample) |
| RU (1) | RU2704619C2 (enExample) |
| SG (3) | SG10201910844SA (enExample) |
| SI (2) | SI3126499T1 (enExample) |
| SM (2) | SMT202000456T1 (enExample) |
| WO (1) | WO2015153800A2 (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI3126499T1 (sl) * | 2014-04-01 | 2020-09-30 | Biogen Ma Inc. | Sestave za moduliranje izražanja SOD-1 |
| EP3174981B1 (en) * | 2014-07-31 | 2020-03-25 | Association Institut de Myologie | Treatment of amyotrophic lateral sclerosis |
| CN112410339A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| RU2716422C2 (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| CA3252099A1 (en) | 2016-05-18 | 2025-06-05 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| BR112018075667B1 (pt) * | 2016-06-14 | 2024-02-20 | Biogen Ma, Inc | Métodos para separar um oligonucleotídeo alvo de uma mistura contendo o oligonucleotídeo alvo e uma impureza relacionada com o produto |
| EP3475292B1 (en) | 2016-06-24 | 2024-09-18 | Biogen MA Inc. | Synthesis of thiolated oligonucleotides without a capping step |
| JP2020518258A (ja) * | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
| WO2018218219A1 (en) * | 2017-05-26 | 2018-11-29 | University Of Miami | Determining onset of amyotrophic lateral sclerosis |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CN119242711A (zh) | 2017-10-16 | 2025-01-03 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| US11474113B2 (en) | 2018-01-25 | 2022-10-18 | Biosen MA Inc. | Methods of treating spinal muscular atrophy |
| TWI833770B (zh) | 2018-06-27 | 2024-03-01 | 美商Ionis製藥公司 | 用於減少 lrrk2 表現之化合物及方法 |
| MX2021005886A (es) * | 2018-11-21 | 2021-06-23 | Ionis Pharmaceuticals Inc | Compuestos y metodos para reducir la expresion de priones. |
| CN113365640A (zh) * | 2018-12-06 | 2021-09-07 | 比奥根Ma公司 | 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白 |
| UY38506A (es) * | 2018-12-14 | 2020-06-30 | Biogen Ma Inc | Composiciones y metodos para tratar y prevenir la esclerosis lateral amiotrofica |
| KR20210153632A (ko) * | 2019-03-26 | 2021-12-17 | 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 | 신경변성 장애를 치료하기 위한 조성물 및 방법 |
| KR20220027059A (ko) * | 2019-05-01 | 2022-03-07 | 블랙 스완 파마슈티컬스, 인코포레이티드 | Sod1 관련 질환의 치료 |
| US11629347B2 (en) | 2019-05-06 | 2023-04-18 | University Of Massachusetts | Anti-C9ORF72 oligonucleotides and related methods |
| KR20220007104A (ko) | 2019-05-08 | 2022-01-18 | 바이오젠 엠에이 인코포레이티드 | 올리고뉴클레오타이드의 수렴형 액상 합성 |
| PL3976791T3 (pl) * | 2019-05-28 | 2025-08-04 | Ionis Pharmaceuticals, Inc. | Związki i sposoby do zmniejszania ekspresji fus |
| WO2022174000A2 (en) | 2021-02-12 | 2022-08-18 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| CA3214585A1 (en) | 2021-03-31 | 2022-10-06 | Biogen Ma Inc. | Treatment of amyotrophic lateral sclerosis |
| TW202304473A (zh) | 2021-04-01 | 2023-02-01 | 美商百健Ma公司 | 向中樞神經系統之核酸遞送 |
| EP4430191A2 (en) * | 2021-11-08 | 2024-09-18 | University Of Massachusetts | Oligonucleotides for sod1 modulation |
| CN114369130B (zh) * | 2021-12-28 | 2023-10-03 | 杭州天龙药业有限公司 | 修饰的硫代寡核苷酸及其应用 |
| US11912997B2 (en) | 2022-06-15 | 2024-02-27 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of Superoxide Dismutase 1 (SOD1), compositions thereof, and methods of use |
| KR20240038469A (ko) | 2022-09-16 | 2024-03-25 | 김정훈 | 사용자 콘텐츠 기반 홍보 서비스 제공 장치 및 방법 |
| CN120202298A (zh) * | 2023-08-29 | 2025-06-24 | 石药集团中奇制药技术(石家庄)有限公司 | 一种抑制sod1基因表达的dsRNA分子及其应用 |
| WO2025126158A2 (en) * | 2023-12-15 | 2025-06-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising small nuclear rna (snrna) targeting sod1 |
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| FR2567892B1 (fr) | 1984-07-19 | 1989-02-17 | Centre Nat Rech Scient | Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons |
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