JP2013226147A5 - - Google Patents
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- JP2013226147A5 JP2013226147A5 JP2013115737A JP2013115737A JP2013226147A5 JP 2013226147 A5 JP2013226147 A5 JP 2013226147A5 JP 2013115737 A JP2013115737 A JP 2013115737A JP 2013115737 A JP2013115737 A JP 2013115737A JP 2013226147 A5 JP2013226147 A5 JP 2013226147A5
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Claims (12)
12〜35ヌクレオチドの長さで、ヒトハンチンチンをコードする核酸を標的とし、SEQ ID NO: 4に対して95%相補的であり、そしてヒトハンチンチンの発現を阻害する、一本鎖アンチセンスオリゴヌクレオチドを含む、ヒトにおいてハンチントン病を治療するための医薬組成物。
アンチセンスオリゴヌクレオチドが、SEQ ID NO: 4に対して100%相補的である、請求項1に記載の医薬組成物。
アンチセンスオリゴヌクレオチドが、17〜25ヌクレオチドの長さ、19〜23ヌクレオチドの長さ、または20ヌクレオチドの長さ、である、請求項1または2に記載の医薬組成物。
少なくとも1つの修飾されたヌクレオシド間結合、糖部分、または核酸塩基を有する、請求項1〜3のいずれか1項の医薬組成物。
5'ウィングセグメントと3'ウィングセグメントとの間に位置するギャップセグメントを有するキメラオリゴヌクレオチドを含む、請求項1〜4のいずれか1項の医薬組成物。
キメラオリゴヌクレオチドのギャップセグメントが2'-デオキシヌクレオチドを含み、そしてウィングセグメントが修飾された糖部分を有するヌクレオチドを含む、請求項5の医薬組成物。
修飾された糖部分が2'-MOE、2'-OMe、または二環式核酸である、請求項6の医薬組成物。
キメラオリゴヌクレオチドのギャップセグメントが10個の2'-デオキシヌクレオチドからなり、また、各ウィングセグメントが5個の2'-O-メトキシエチル-修飾ヌクレオチドからなる、請求項6の医薬組成物。
少なくとも1個の修飾されたヌクレオシド間結合を有する、請求項4の医薬組成物。
各ヌクレオシド間結合が、ホスホロチオエートヌクレオシド間結合である、請求項9の医薬組成物。
各シトシンが、5-メチルシトシンである、請求項4の医薬組成物。
20ヌクレオチドの長さであり、5'ウィングセグメントと3'ウィングセグメントとの間に位置するギャップセグメントを有するキメラオリゴヌクレオチドを含み、キメラオリゴヌクレオチドのギャップセグメントが10個の2'-デオキシヌクレオチドからなり、そして各ウィングセグメントが5個の2'-O-メトキシエチル-修飾ヌクレオチドからなる、請求項1〜3のいずれか1項の医薬組成物。
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US76295406P | 2006-01-26 | 2006-01-26 | |
US60/762,954 | 2006-01-26 | ||
US83629006P | 2006-08-07 | 2006-08-07 | |
US60/836,290 | 2006-08-07 |
Related Parent Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2008552447A Division JP5425474B2 (ja) | 2006-01-26 | 2007-01-26 | ハンチンチン対する、組成物及びその使用 |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2015252875A Division JP6395058B2 (ja) | 2006-01-26 | 2015-12-25 | ハンチンチン対する、組成物及びその使用 |
Publications (3)
Publication Number | Publication Date |
---|---|
JP2013226147A JP2013226147A (ja) | 2013-11-07 |
JP2013226147A5 true JP2013226147A5 (ja) | 2014-03-13 |
JP5916660B2 JP5916660B2 (ja) | 2016-05-11 |
Family
ID=38164262
Family Applications (5)
Application Number | Title | Priority Date | Filing Date |
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JP2008552447A Active JP5425474B2 (ja) | 2006-01-26 | 2007-01-26 | ハンチンチン対する、組成物及びその使用 |
JP2013115737A Active JP5916660B2 (ja) | 2006-01-26 | 2013-05-31 | ハンチンチン対する、組成物及びその使用 |
JP2015252875A Active JP6395058B2 (ja) | 2006-01-26 | 2015-12-25 | ハンチンチン対する、組成物及びその使用 |
JP2018061748A Pending JP2018117635A (ja) | 2006-01-26 | 2018-03-28 | ハンチンチン対する、組成物及びその使用 |
JP2020023793A Pending JP2020096624A (ja) | 2006-01-26 | 2020-02-14 | ハンチンチン対する、組成物及びその使用 |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
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JP2008552447A Active JP5425474B2 (ja) | 2006-01-26 | 2007-01-26 | ハンチンチン対する、組成物及びその使用 |
Family Applications After (3)
Application Number | Title | Priority Date | Filing Date |
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JP2015252875A Active JP6395058B2 (ja) | 2006-01-26 | 2015-12-25 | ハンチンチン対する、組成物及びその使用 |
JP2018061748A Pending JP2018117635A (ja) | 2006-01-26 | 2018-03-28 | ハンチンチン対する、組成物及びその使用 |
JP2020023793A Pending JP2020096624A (ja) | 2006-01-26 | 2020-02-14 | ハンチンチン対する、組成物及びその使用 |
Country Status (12)
Country | Link |
---|---|
US (14) | US7951934B2 (ja) |
EP (5) | EP1991677A2 (ja) |
JP (5) | JP5425474B2 (ja) |
AU (4) | AU2007210038B2 (ja) |
CA (1) | CA2638908C (ja) |
DK (1) | DK2161038T3 (ja) |
ES (2) | ES2744098T3 (ja) |
HK (1) | HK1141449A1 (ja) |
PL (2) | PL2161038T3 (ja) |
PT (1) | PT2161038E (ja) |
SI (1) | SI2161038T1 (ja) |
WO (2) | WO2007089584A2 (ja) |
Families Citing this family (89)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2007089584A2 (en) | 2006-01-26 | 2007-08-09 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to huntingtin |
CA2660523C (en) | 2006-08-11 | 2019-03-19 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
EP2410053B2 (en) | 2006-10-18 | 2020-07-15 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
US20090036395A1 (en) | 2007-04-26 | 2009-02-05 | Davidson Beverly L | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
DK2164967T3 (en) | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
EP2167135A2 (en) * | 2007-07-12 | 2010-03-31 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
HUE028662T2 (en) | 2007-10-26 | 2016-12-28 | Academisch Ziekenhuis Leiden | Preparations and methods for controlling muscle disorders |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
WO2009099326A1 (en) | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
US8679750B2 (en) | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
JP2011529686A (ja) | 2008-07-29 | 2011-12-15 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | ポリグルタミンタンパク質発現の選択的阻害 |
US20110237646A1 (en) * | 2008-08-07 | 2011-09-29 | Isis Pharmaceuticals, Inc. | Modulation of transthyretin expression for the treatment of cns related disorders |
NZ603603A (en) * | 2008-10-15 | 2014-06-27 | Isis Pharmaceuticals Inc | Modulation of factor 11 expression |
JP5645840B2 (ja) | 2008-12-02 | 2014-12-24 | 株式会社Wave Life Sciences Japan | リン原子修飾核酸の合成方法 |
WO2010123369A1 (en) | 2009-04-24 | 2010-10-28 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
CA2762369C (en) * | 2009-05-18 | 2021-12-28 | Joseph Collard | Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor |
KR20190037378A (ko) * | 2009-06-17 | 2019-04-05 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 smn2 스플라이싱을 조정하기 위한 조성물 및 방법 |
CN102596204B (zh) | 2009-07-06 | 2016-11-23 | 波涛生命科学有限公司 | 新的核酸前药及其使用方法 |
CN102625809B (zh) | 2009-09-11 | 2015-06-24 | Isis制药公司 | 亨廷顿表达的调节 |
WO2011078672A1 (en) | 2009-12-24 | 2011-06-30 | Prosensa Technologies B.V. | Molecule for treating an inflammatory disorder |
WO2011097614A1 (en) * | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Mehods and compositions useful in diseases or conditions related to repeat expansion |
WO2011097641A1 (en) * | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
US9006198B2 (en) * | 2010-02-08 | 2015-04-14 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
DK2534248T3 (en) | 2010-02-08 | 2018-11-19 | Ionis Pharmaceuticals Inc | SELECTIVE REDUCTION OF ALLELVARIANS |
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 |
DK2563920T3 (en) | 2010-04-29 | 2017-05-22 | Ionis Pharmaceuticals Inc | Modulation of transthyretin expression |
JP5981428B2 (ja) * | 2010-07-19 | 2016-08-31 | 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Ionis Pharmaceuticals,Inc. | 筋緊張性ジストロフィー・プロテインキナーゼ(dmpk)発現の調整 |
WO2012018257A1 (en) * | 2010-08-05 | 2012-02-09 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins |
EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
US9663783B2 (en) * | 2010-11-17 | 2017-05-30 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
WO2012109395A1 (en) | 2011-02-08 | 2012-08-16 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
ES2739804T3 (es) | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
PT3505528T (pt) | 2011-04-21 | 2021-02-23 | Glaxo Group Ltd | Modulação da expressão do vírus da hepatite b (vhb) |
EP2699269A1 (en) | 2011-04-22 | 2014-02-26 | Prosensa Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1) |
JP6128529B2 (ja) | 2011-07-19 | 2017-05-17 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | 官能化核酸の合成のための方法 |
WO2013022984A1 (en) | 2011-08-11 | 2013-02-14 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
WO2013033223A1 (en) | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
CA2862628C (en) | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
WO2013159108A2 (en) * | 2012-04-20 | 2013-10-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
SI2841578T1 (sl) | 2012-04-23 | 2017-12-29 | Biomarin Technologies B.V. | Rna modulirajoči oligonukleotidi z izboljšanimi karakteristikami za zdravljenje nevromuskularnih motenj |
PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
AU2013287630B2 (en) | 2012-07-13 | 2017-05-25 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
WO2014012081A2 (en) | 2012-07-13 | 2014-01-16 | Ontorii, Inc. | Chiral control |
EP2906699A4 (en) | 2012-10-11 | 2016-06-08 | Ionis Pharmaceuticals Inc | OLIGOMER COMPOUNDS WITH BICYCLIC NUCLEOSIDES AND USES THEREOF |
WO2014059356A2 (en) | 2012-10-12 | 2014-04-17 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
WO2014062691A2 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
JP6387084B2 (ja) * | 2013-05-01 | 2018-09-05 | 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Ionis Pharmaceuticals,Inc. | アポリポタンパク質c−iiiの発現を調節するための組成物および方法 |
GB201308259D0 (en) * | 2013-05-08 | 2013-06-12 | Iles Raymond K | Cancer diagnosis and monitoring |
US10435430B2 (en) | 2013-07-31 | 2019-10-08 | Ionis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
WO2015054676A2 (en) | 2013-10-11 | 2015-04-16 | Isis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
US10160969B2 (en) | 2014-01-16 | 2018-12-25 | Wave Life Sciences Ltd. | Chiral design |
AU2015264263B2 (en) | 2014-05-20 | 2021-08-05 | University Of Iowa Research Foundation | Huntington's disease therapeutic compounds |
GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
GB201504124D0 (en) | 2015-03-11 | 2015-04-22 | Proqr Therapeutics B V | Oligonucleotides |
CN113265404A (zh) | 2015-04-16 | 2021-08-17 | Ionis制药公司 | 用于调节c9orf72表达的组合物 |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
JP6923517B2 (ja) | 2015-10-09 | 2021-08-18 | ユニバーシティ・オブ・サザンプトン | 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング |
EP3359662A4 (en) * | 2015-10-09 | 2019-06-19 | The Children's Hospital of Philadelphia | COMPOSITIONS AND METHODS OF TREATING HUNTINGTON DISEASE AND RELATED DISEASES |
WO2017079291A1 (en) | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
ES2879995T3 (es) | 2015-12-10 | 2021-11-23 | Ptc Therapeutics Inc | Métodos para el tratamiento de la enfermedad de Huntington |
JP7049248B2 (ja) | 2015-12-14 | 2022-04-06 | コールド スプリング ハーバー ラボラトリー | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
JP2019535839A (ja) | 2016-11-29 | 2019-12-12 | ピュアテック ヘルス エルエルシー | 治療剤の送達のためのエクソソーム |
AU2018282154B2 (en) | 2017-06-05 | 2022-04-07 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
WO2019005980A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | METHODS OF TREATING HUNTINGTON'S DISEASE |
BR112019027719A2 (pt) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics, Inc. | métodos para tratar a doença de huntington |
SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
TWI809004B (zh) * | 2017-11-09 | 2023-07-21 | 美商Ionis製藥公司 | 用於降低snca表現之化合物及方法 |
JP2021502882A (ja) | 2017-11-15 | 2021-02-04 | アルキオーネ・ライフサイエンシズ・インコーポレイテッドAlcyone Lifesciences, Inc. | 治療法別の事前プログラムされた自動注射デバイス |
PE20201501A1 (es) | 2018-01-12 | 2020-12-29 | Bristol Myers Squibb Co | Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos |
KR20210005559A (ko) | 2018-03-27 | 2021-01-14 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
AU2019294478B2 (en) | 2018-06-27 | 2023-03-23 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating Huntington's disease |
MX2022005254A (es) | 2019-11-01 | 2022-06-29 | Novartis Ag | El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington. |
US20230340470A1 (en) * | 2019-12-20 | 2023-10-26 | Asklepios Biopharmaceutical, Inc. | Methods for treating huntington's disease |
AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
TW202305134A (zh) | 2021-03-29 | 2023-02-01 | 美商艾拉倫製藥股份有限公司 | 杭丁頓蛋白(HTT)iRNA劑組成物及其使用方法 |
WO2023034870A2 (en) | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
TW202334418A (zh) * | 2021-10-29 | 2023-09-01 | 美商艾拉倫製藥股份有限公司 | 杭丁頓(HTT)iRNA劑組成物及其使用方法 |
WO2023168304A2 (en) * | 2022-03-02 | 2023-09-07 | Triplet Therapeutics, Inc. | Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity |
WO2024077267A1 (en) * | 2022-10-07 | 2024-04-11 | The Broad Institute, Inc. | Prime editing methods and compositions for treating triplet repeat disorders |
Family Cites Families (121)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US667046A (en) | 1899-06-23 | 1901-01-29 | John W Weeks | Pressure-reducing valve. |
US2526893A (en) | 1946-08-28 | 1950-10-24 | Cutler Hammer Inc | Dynamic braking control |
US4426330A (en) | 1981-07-20 | 1984-01-17 | Lipid Specialties, Inc. | Synthetic phospholipid compounds |
US4534899A (en) | 1981-07-20 | 1985-08-13 | Lipid Specialties, Inc. | Synthetic phospholipid compounds |
US5118800A (en) | 1983-12-20 | 1992-06-02 | California Institute Of Technology | Oligonucleotides possessing a primary amino group in the terminal nucleotide |
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 |
US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
DE3788914T2 (de) | 1986-09-08 | 1994-08-25 | Ajinomoto Kk | Verbindungen zur Spaltung von RNS an eine spezifische Position, Oligomere, verwendet bei der Herstellung dieser Verbindungen und Ausgangsprodukte für die Synthese dieser Oligomere. |
ATE151467T1 (de) | 1987-11-30 | 1997-04-15 | Univ Iowa Res Found | Durch modifikationen an der 3'-terminalen phosphodiesterbindung stabilisierte dna moleküle, ihre verwendung als nukleinsäuresonden sowie als therapeutische mittel zur hemmung der expression spezifischer zielgene |
US5403711A (en) | 1987-11-30 | 1995-04-04 | University Of Iowa Research Foundation | Nucleic acid hybridization and amplification method for detection of specific sequences in which a complementary labeled nucleic acid probe is cleaved |
US5354844A (en) | 1989-03-16 | 1994-10-11 | Boehringer Ingelheim International Gmbh | Protein-polycation conjugates |
US5108921A (en) | 1989-04-03 | 1992-04-28 | Purdue Research Foundation | Method for enhanced transmembrane transport of exogenous molecules |
US5256775A (en) | 1989-06-05 | 1993-10-26 | Gilead Sciences, Inc. | Exonuclease-resistant oligonucleotides |
US5227170A (en) | 1989-06-22 | 1993-07-13 | Vestar, Inc. | Encapsulation process |
US5591722A (en) | 1989-09-15 | 1997-01-07 | Southern Research Institute | 2'-deoxy-4'-thioribonucleosides and their antiviral activity |
US5013556A (en) | 1989-10-20 | 1991-05-07 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
US5356633A (en) | 1989-10-20 | 1994-10-18 | Liposome Technology, Inc. | Method of treatment of inflamed tissues |
US5527528A (en) | 1989-10-20 | 1996-06-18 | Sequus Pharmaceuticals, Inc. | Solid-tumor treatment method |
EP0942000B1 (en) | 1989-10-24 | 2004-06-23 | Isis Pharmaceuticals, Inc. | 2'-Modified oligonucleotides |
US5580575A (en) | 1989-12-22 | 1996-12-03 | Imarx Pharmaceutical Corp. | Therapeutic drug delivery systems |
US5469854A (en) | 1989-12-22 | 1995-11-28 | Imarx Pharmaceutical Corp. | Methods of preparing gas-filled liposomes |
US5670633A (en) | 1990-01-11 | 1997-09-23 | Isis Pharmaceuticals, Inc. | Sugar modified oligonucleotides that detect and modulate gene expression |
US5646265A (en) | 1990-01-11 | 1997-07-08 | Isis Pharmceuticals, Inc. | Process for the preparation of 2'-O-alkyl purine phosphoramidites |
US5623065A (en) | 1990-08-13 | 1997-04-22 | Isis Pharmaceuticals, Inc. | Gapped 2' modified oligonucleotides |
US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
US5220007A (en) | 1990-02-15 | 1993-06-15 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of RNA and production of encoded polypeptides |
US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
GB9009980D0 (en) | 1990-05-03 | 1990-06-27 | Amersham Int Plc | Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides |
EP0745689A3 (en) | 1990-05-11 | 1996-12-11 | Microprobe Corporation | A dipstick for a nucleic acid hybridization assay |
US6582908B2 (en) | 1990-12-06 | 2003-06-24 | Affymetrix, Inc. | Oligonucleotides |
JP3220180B2 (ja) | 1991-05-23 | 2001-10-22 | 三菱化学株式会社 | 薬剤含有タンパク質結合リポソーム |
NZ244306A (en) | 1991-09-30 | 1995-07-26 | Boehringer Ingelheim Int | Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation |
US5521291A (en) | 1991-09-30 | 1996-05-28 | Boehringer Ingelheim International, Gmbh | Conjugates for introducing nucleic acid into higher eucaryotic cells |
DE59208572D1 (de) | 1991-10-17 | 1997-07-10 | Ciba Geigy Ag | Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte |
US5359044A (en) | 1991-12-13 | 1994-10-25 | Isis Pharmaceuticals | Cyclobutyl oligonucleotide surrogates |
US5700922A (en) * | 1991-12-24 | 1997-12-23 | Isis Pharmaceuticals, Inc. | PNA-DNA-PNA chimeric macromolecules |
FR2687679B1 (fr) | 1992-02-05 | 1994-10-28 | Centre Nat Rech Scient | Oligothionucleotides. |
FR2692265B1 (fr) | 1992-05-25 | 1996-11-08 | Centre Nat Rech Scient | Composes biologiquement actifs de type phosphotriesters. |
EP0577558A2 (de) | 1992-07-01 | 1994-01-05 | Ciba-Geigy Ag | Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte |
US5652355A (en) | 1992-07-23 | 1997-07-29 | Worcester Foundation For Experimental Biology | Hybrid oligonucleotide phosphorothioates |
US6180403B1 (en) * | 1999-10-28 | 2001-01-30 | Isis Pharmaceuticals Inc. | Antisense inhibition of tumor necrosis factor alpha converting enzyme (TACE) expression |
US5583020A (en) | 1992-11-24 | 1996-12-10 | Ribozyme Pharmaceuticals, Inc. | Permeability enhancers for negatively charged polynucleotides |
JP3351476B2 (ja) | 1993-01-22 | 2002-11-25 | 三菱化学株式会社 | リン脂質誘導体及びそれを含有するリポソーム |
US5395619A (en) | 1993-03-03 | 1995-03-07 | Liposome Technology, Inc. | Lipid-polymer conjugates and liposomes |
ATE155467T1 (de) | 1993-03-30 | 1997-08-15 | Sanofi Sa | Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen |
DE4311944A1 (de) | 1993-04-10 | 1994-10-13 | Degussa | Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen |
US5462854A (en) | 1993-04-19 | 1995-10-31 | Beckman Instruments, Inc. | Inverse linkage oligonucleotides for chemical and enzymatic processes |
FR2705099B1 (fr) | 1993-05-12 | 1995-08-04 | Centre Nat Rech Scient | Oligonucléotides phosphorothioates triesters et procédé de préparation. |
US5534259A (en) | 1993-07-08 | 1996-07-09 | Liposome Technology, Inc. | Polymer compound and coated particle composition |
US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
US5417978A (en) | 1993-07-29 | 1995-05-23 | Board Of Regents, The University Of Texas System | Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use |
US5801154A (en) | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
US5446137B1 (en) | 1993-12-09 | 1998-10-06 | Behringwerke Ag | Oligonucleotides containing 4'-substituted nucleotides |
ATE196311T1 (de) | 1993-12-09 | 2000-09-15 | Univ Jefferson | Verbindungen und verfahren zur ortsspezifischen mutation in eukaryotischen zellen |
US5595756A (en) | 1993-12-22 | 1997-01-21 | Inex Pharmaceuticals Corporation | Liposomal compositions for enhanced retention of bioactive agents |
US5519134A (en) | 1994-01-11 | 1996-05-21 | Isis Pharmaceuticals, Inc. | Pyrrolidine-containing monomers and oligomers |
US5627053A (en) | 1994-03-29 | 1997-05-06 | Ribozyme Pharmaceuticals, Inc. | 2'deoxy-2'-alkylnucleotide containing nucleic acid |
US5543152A (en) | 1994-06-20 | 1996-08-06 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
US5597909A (en) | 1994-08-25 | 1997-01-28 | Chiron Corporation | Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use |
US5591721A (en) | 1994-10-25 | 1997-01-07 | Hybridon, Inc. | Method of down-regulating gene expression |
US5512295A (en) | 1994-11-10 | 1996-04-30 | The Board Of Trustees Of The Leland Stanford Junior University | Synthetic liposomes for enhanced uptake and delivery |
US5792747A (en) | 1995-01-24 | 1998-08-11 | The Administrators Of The Tulane Educational Fund | Highly potent agonists of growth hormone releasing hormone |
US5652356A (en) | 1995-08-17 | 1997-07-29 | Hybridon, Inc. | Inverted chimeric and hybrid oligonucleotides |
US20040266706A1 (en) * | 2002-11-05 | 2004-12-30 | Muthiah Manoharan | Cross-linked oligomeric compounds and their use in gene modulation |
US6368855B1 (en) * | 1996-06-11 | 2002-04-09 | Antigen Express, Inc. | MHC class II antigen presenting cells containing oligonucleotides which inhibit Ii protein expression |
ATE232880T1 (de) | 1996-11-18 | 2003-03-15 | Takeshi Imanishi | Neue nucleotidanaloga |
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 |
US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
US20030228597A1 (en) | 1998-04-13 | 2003-12-11 | Cowsert Lex M. | Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation |
US20030096775A1 (en) * | 2001-10-23 | 2003-05-22 | Isis Pharmaceuticals Inc. | Antisense modulation of complement component C3 expression |
US5998148A (en) | 1999-04-08 | 1999-12-07 | Isis Pharmaceuticals Inc. | Antisense modulation of microtubule-associated protein 4 expression |
US6147200A (en) | 1999-08-19 | 2000-11-14 | Isis Pharmaceuticals, Inc. | 2'-O-acetamido modified monomers and oligomers |
US6287860B1 (en) | 2000-01-20 | 2001-09-11 | Isis Pharmaceuticals, Inc. | Antisense inhibition of MEKK2 expression |
AU4819101A (en) * | 2000-04-13 | 2001-10-30 | University Of British Columbia, The | Modulating cell survival by modulating huntingtin function |
AU2001296412A1 (en) * | 2000-09-29 | 2002-04-08 | Isis Pharmaceuticals, Inc. | Antisense modulation of mekk4 expression |
US20050191638A1 (en) * | 2002-02-20 | 2005-09-01 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
CA2455424A1 (en) | 2001-08-07 | 2003-02-20 | University Of Delaware | Compositions and methods for the prevention and treatment of huntington's disease |
US20040096880A1 (en) | 2001-08-07 | 2004-05-20 | Kmiec Eric B. | Compositions and methods for the treatment of diseases exhibiting protein misassembly and aggregation |
US7176303B2 (en) | 2003-11-06 | 2007-02-13 | Isis Pharmaceuticals, Inc. | Modulation of STAT5 expression |
ATE443133T1 (de) | 2002-02-01 | 2009-10-15 | Life Technologies Corp | Oligonukleotidzusammensetzungen mit verbesserter effizienz |
US20050096284A1 (en) * | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
US20030232443A1 (en) * | 2002-06-18 | 2003-12-18 | Isis Pharmaceuticals Inc. | Antisense modulation of centromere protein B expression |
US20040092465A1 (en) * | 2002-11-11 | 2004-05-13 | Isis Pharmaceuticals Inc. | Modulation of huntingtin interacting protein 1 expression |
US20040102398A1 (en) | 2002-11-23 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of B7H expression |
US20050255086A1 (en) * | 2002-08-05 | 2005-11-17 | Davidson Beverly L | Nucleic acid silencing of Huntington's Disease gene |
US20080274989A1 (en) * | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
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 |
AU2003273336A1 (en) | 2002-09-18 | 2004-04-08 | Isis Pharmaceuticals, Inc. | Efficient reduction of target rna's by single- and double-stranded oligomeric compounds |
US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
EP1560931B1 (en) * | 2002-11-14 | 2011-07-27 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
US7250289B2 (en) * | 2002-11-20 | 2007-07-31 | Affymetrix, Inc. | Methods of genetic analysis of mouse |
US7217807B2 (en) * | 2002-11-26 | 2007-05-15 | Rosetta Genomics Ltd | Bioinformatically detectable group of novel HIV regulatory genes and uses thereof |
US20040191818A1 (en) * | 2003-02-26 | 2004-09-30 | O'toole Margot Mary | Compositions and methods for diagnosing and treating autoimmune diseases |
EP1605978B1 (en) | 2003-03-07 | 2010-09-01 | Alnylam Pharmaceuticals Inc. | Therapeutic compositions |
CA2526893C (en) | 2003-05-14 | 2010-10-26 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
EP1646372A2 (en) * | 2003-07-14 | 2006-04-19 | Decode Genetics EHF. | Methods of diagnosis and treatment for asthma based on haplotype association |
ES2808561T3 (es) | 2003-09-12 | 2021-03-01 | Univ Massachusetts | Interferencia por ARN para el tratamiento de trastornos de ganancia de función |
WO2005045032A2 (en) | 2003-10-20 | 2005-05-19 | Sima Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF EARLY GROWTH RESPONSE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US7341835B2 (en) * | 2004-01-13 | 2008-03-11 | Affymetrix, Inc. | Methods of analysis of alternative splicing in mouse |
WO2005083436A1 (en) * | 2004-02-26 | 2005-09-09 | Bayer Healthcare Ag | Diagnostics and therapeutics for diseases associated with integrin-linked kinase 1 (ilk1) |
EP1735443A2 (en) * | 2004-04-14 | 2006-12-27 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US7374927B2 (en) * | 2004-05-03 | 2008-05-20 | Affymetrix, Inc. | Methods of analysis of degraded nucleic acid samples |
AU2005248147A1 (en) * | 2004-05-11 | 2005-12-08 | Alphagen Co., Ltd. | Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same |
US7323308B2 (en) * | 2004-09-03 | 2008-01-29 | Affymetrix, Inc. | Methods of genetic analysis of E. coli |
EP1891217A2 (en) | 2005-05-27 | 2008-02-27 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of stromal cell-derived factor-1 (sdf-1) gene expression using short interfering nucleic acid (sina) |
DE202005008426U1 (de) | 2005-05-31 | 2005-09-15 | Ricon Sieb Und Foerdertechnik | Fördervorrichtung mit Fördertuch |
EP1937066A4 (en) | 2005-08-18 | 2008-12-24 | Alnylam Pharmaceuticals Inc | METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES |
WO2007051045A2 (en) | 2005-10-28 | 2007-05-03 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of huntingtin gene |
US8658608B2 (en) * | 2005-11-23 | 2014-02-25 | Yale University | Modified triple-helix forming oligonucleotides for targeted mutagenesis |
WO2007089584A2 (en) | 2006-01-26 | 2007-08-09 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to huntingtin |
US7901882B2 (en) * | 2006-03-31 | 2011-03-08 | Affymetrix, Inc. | Analysis of methylation using nucleic acid arrays |
WO2007120883A2 (en) | 2006-04-12 | 2007-10-25 | Isis Pharmaceuticals, Inc. | Compositions and their uses directed to hepcidin |
CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
CA2660523C (en) | 2006-08-11 | 2019-03-19 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
US20080039415A1 (en) | 2006-08-11 | 2008-02-14 | Gregory Robert Stewart | Retrograde transport of sirna and therapeutic uses to treat neurologic disorders |
WO2009023855A2 (en) * | 2007-08-15 | 2009-02-19 | Isis Pharmaceuticals, Inc. | Tetrahydropyran nucleic acid analogs |
AU2010229872A1 (en) | 2009-03-26 | 2011-11-03 | The Regents Of The University Of California | Mesenchymal stem cells producing inhibitory RNA for disease modification |
US8957038B2 (en) | 2009-07-15 | 2015-02-17 | Medtronic, Inc. | Treatment of neurological disorders |
US9574191B2 (en) | 2010-02-03 | 2017-02-21 | The Board Of Regents Of The University Of Texas System | Selective inhibition of polyglutamine protein expression |
US10867398B2 (en) | 2017-11-21 | 2020-12-15 | Reliance Core Consulting LLC | Methods, systems, apparatuses and devices for facilitating motion analysis in an environment |
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