CY1123367T1 - Μεθοδοι και συνθεσεις για τη ρυθμιση της εκφρασης της απολιποπρωτεϊνης (α) - Google Patents
Μεθοδοι και συνθεσεις για τη ρυθμιση της εκφρασης της απολιποπρωτεϊνης (α)Info
- Publication number
- CY1123367T1 CY1123367T1 CY20201100915T CY201100915T CY1123367T1 CY 1123367 T1 CY1123367 T1 CY 1123367T1 CY 20201100915 T CY20201100915 T CY 20201100915T CY 201100915 T CY201100915 T CY 201100915T CY 1123367 T1 CY1123367 T1 CY 1123367T1
- Authority
- CY
- Cyprus
- Prior art keywords
- methods
- disorders
- diseases
- apolypoprotein
- regulating
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
- 230000001105 regulatory effect Effects 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 7
- 102100040214 Apolipoprotein(a) Human genes 0.000 abstract 4
- 101710115418 Apolipoprotein(a) Proteins 0.000 abstract 4
- 208000035475 disorder Diseases 0.000 abstract 4
- 201000010099 disease Diseases 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 2
- 208000024172 Cardiovascular disease Diseases 0.000 abstract 1
- 230000002526 effect on cardiovascular system Effects 0.000 abstract 1
- 208000027866 inflammatory disease Diseases 0.000 abstract 1
- 230000002757 inflammatory effect Effects 0.000 abstract 1
- 208000030159 metabolic disease Diseases 0.000 abstract 1
- 230000002265 prevention Effects 0.000 abstract 1
Classifications
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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
- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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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
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21007—Plasmin (3.4.21.7), i.e. fibrinolysin
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- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- 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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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/34—Spatial arrangement of the modifications
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- 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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- 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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- 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
- Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
- Y02P20/00—Technologies relating to chemical industry
- Y02P20/50—Improvements relating to the production of bulk chemicals
- Y02P20/55—Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Diabetes (AREA)
- Urology & Nephrology (AREA)
- Vascular Medicine (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Toxicology (AREA)
- Pain & Pain Management (AREA)
- Rheumatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Στο παρόν αποκαλύπτονται αντινοηματικές ενώσεις και μέθοδοι για τη μείωση της apo(a) για τη θεραπευτική αγωγή, πρόληψη, βελτίωση ασθενειών, διαταραχών ή καταστάσεων που συνδέονται με apo{a) ή Lp(a). Ορισμένες ασθένειες, διαταραχές ή καταστάσεις που συνδέονται με apo(a) ή Lp(a) περιλαμβάνουν φλεγμονώδεις, 10 καρδιαγγειακές ή/και μεταβολικές ασθένειες, διαταραχές ή καταστάσεις. Οι αντινοηματικές ενώσεις που αποκαλύπτονται στο παρόν μπορούν να χρησιμοποιηθούν για τη θεραπευτική αγωγή τέτοιων ασθενειών, διαταραχών ή καταστάσεων σε ένα άτομο που χρήζει αυτής.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261651539P | 2012-05-24 | 2012-05-24 | |
| PCT/US2013/042532 WO2013177468A2 (en) | 2012-05-24 | 2013-05-23 | Methods and compositions for modulating apolipoprotein(a) expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CY1123367T1 true CY1123367T1 (el) | 2021-12-31 |
Family
ID=49624530
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20201100915T CY1123367T1 (el) | 2012-05-24 | 2020-09-29 | Μεθοδοι και συνθεσεις για τη ρυθμιση της εκφρασης της απολιποπρωτεϊνης (α) |
Country Status (21)
| Country | Link |
|---|---|
| US (3) | US20150152411A1 (el) |
| EP (3) | EP2855500B1 (el) |
| JP (3) | JP6082809B2 (el) |
| KR (4) | KR20170142997A (el) |
| CN (2) | CN104302654B (el) |
| AU (4) | AU2013266196A1 (el) |
| CA (2) | CA2874480C (el) |
| CY (1) | CY1123367T1 (el) |
| DK (1) | DK2855500T3 (el) |
| ES (1) | ES2820548T3 (el) |
| HR (1) | HRP20201426T1 (el) |
| HU (1) | HUE051680T2 (el) |
| IL (2) | IL235746B (el) |
| LT (1) | LT2855500T (el) |
| MX (2) | MX388459B (el) |
| NZ (1) | NZ631071A (el) |
| PL (1) | PL2855500T3 (el) |
| PT (1) | PT2855500T (el) |
| RU (2) | RU2748495C2 (el) |
| SI (1) | SI2855500T1 (el) |
| WO (1) | WO2013177468A2 (el) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013173789A2 (en) | 2012-05-17 | 2013-11-21 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide compositions |
| CA3170716A1 (en) * | 2013-02-14 | 2014-08-21 | Ionis Pharmaceuticals, Inc. | Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations |
| KR102482890B1 (ko) | 2013-05-01 | 2022-12-30 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법 |
| SI3137605T1 (sl) | 2014-05-01 | 2021-02-26 | Ionis Pharmaceuticals, Inc. | Sestavki in metode za modulacijo ekspresije angiopoetin 3-podobnega |
| WO2015179693A1 (en) | 2014-05-22 | 2015-11-26 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| JP6606557B2 (ja) * | 2015-04-16 | 2019-11-13 | アイオーニス ファーマシューティカルズ, インコーポレーテッド | C9orf72発現を調節するための組成物 |
| JOP20210043A1 (ar) * | 2015-10-01 | 2017-06-16 | Arrowhead Pharmaceuticals Inc | تراكيب وأساليب لتثبيط تعبير جيني للـ lpa |
| DK4119569T3 (da) | 2015-11-06 | 2024-08-12 | Ionis Pharmaceuticals Inc | Konjugerede antisense-forbindelser til anvendelse i behandling |
| CA2999341A1 (en) | 2015-11-06 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Modulating apolipoprotein (a) expression |
| CN109661233A (zh) | 2016-10-06 | 2019-04-19 | Ionis 制药公司 | 缀合低聚化合物的方法 |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| MY200352A (en) | 2018-05-22 | 2023-12-21 | Ionis Pharmaceuticals Inc | Modulators of apol1 expression |
| EP3877523A1 (en) | 2018-11-09 | 2021-09-15 | Novartis AG | Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a) |
| UA128236C2 (uk) * | 2018-12-21 | 2024-05-15 | Айоніс Фармасутікалз, Інк. | Модулятори експресії hsd17b13 |
| EP4403633A1 (en) * | 2021-09-18 | 2024-07-24 | Genoval Therapeutics Co., Ltd. | Lpa inhibitor and use thereof |
| TW202345865A (zh) * | 2022-01-24 | 2023-12-01 | 大陸商上海舶望製藥有限公司 | 抑制LPA(Apo(a))蛋白表達的組合物和方法 |
| CN114703184B (zh) * | 2022-03-11 | 2024-06-18 | 厦门甘宝利生物医药有限公司 | Lpa抑制剂及其用途 |
| KR20250163949A (ko) * | 2023-03-20 | 2025-11-21 | 버브 테라퓨틱스, 인크. | 심혈관 질환의 치료를 위한 lpa 유전자의 생체내 닉카제-기반 편집 |
| CN118685405A (zh) * | 2023-06-20 | 2024-09-24 | 施能康医药科技(苏州)有限公司 | 靶向脂蛋白a的核酸及其用途 |
| WO2025064821A2 (en) | 2023-09-21 | 2025-03-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for inhibiting lpa |
| CN117568313B (zh) * | 2024-01-15 | 2024-04-26 | 上海贝斯昂科生物科技有限公司 | 基因编辑组合物及其用途 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US2699808A (en) | 1944-10-06 | 1955-01-18 | Mark W Lowe | Apparatus for peeling tomatoes |
| US2699508A (en) | 1951-12-21 | 1955-01-11 | Selectronics Inc | Method of mounting and construction of mounting for low frequency piezoelectric crystals |
| JPH08504559A (ja) | 1992-12-14 | 1996-05-14 | ハネウエル・インコーポレーテッド | 個別に制御される冗長巻線を有するモータシステム |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| US5656408A (en) | 1996-04-29 | 1997-08-12 | Xerox Corporation | Coated carrier particles |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6770748B2 (en) | 1997-03-07 | 2004-08-03 | Takeshi Imanishi | Bicyclonucleoside and oligonucleotide analogue |
| KR100414936B1 (ko) | 1997-09-12 | 2004-01-13 | 엑시콘 에이/에스 | 이환 및 삼환 뉴클레오시드, 뉴클레오타이드 및올리고뉴클레오타이드 동족체 |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| CA2316808C (en) * | 1998-01-08 | 2007-04-10 | Aventis Pharmaceuticals Products Inc. | Transgenic rabbit that expresses a functional human lipoprotein (a) |
| PT1178999E (pt) | 1999-05-04 | 2007-06-26 | Santaris Pharma As | Análogos de l-ribo-lna |
| US6525191B1 (en) | 1999-05-11 | 2003-02-25 | Kanda S. Ramasamy | Conformationally constrained L-nucleosides |
| CA2452458A1 (en) | 2001-07-03 | 2003-01-16 | Isis Pharmaceuticals, Inc. | Nuclease resistant chimeric oligonucleotides |
| US7227014B2 (en) * | 2001-08-07 | 2007-06-05 | Isis Pharmaceuticals, Inc. | Antisense modulation of apolipoprotein (a) expression |
| US7259150B2 (en) * | 2001-08-07 | 2007-08-21 | Isis Pharmaceuticals, Inc. | Modulation of apolipoprotein (a) expression |
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