HRP20201200T1 - OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK - Google Patents

OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK Download PDF

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Publication number
HRP20201200T1
HRP20201200T1 HRP20201200TT HRP20201200T HRP20201200T1 HR P20201200 T1 HRP20201200 T1 HR P20201200T1 HR P20201200T T HRP20201200T T HR P20201200TT HR P20201200 T HRP20201200 T HR P20201200T HR P20201200 T1 HRP20201200 T1 HR P20201200T1
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HR
Croatia
Prior art keywords
rna molecule
vector
expression
use according
htt gene
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HRP20201200TT
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English (en)
Croatian (hr)
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Anastasia Khvorova
Neil Aronin
Julia ALTERMAN
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University Of Massachusetts
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Publication of HRP20201200T1 publication Critical patent/HRP20201200T1/hr

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    • C12N15/09—Recombinant DNA-technology
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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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HRP20201200TT 2015-04-03 2016-04-01 OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK HRP20201200T1 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562142731P 2015-04-03 2015-04-03
US201662289274P 2016-01-31 2016-01-31
PCT/US2016/025722 WO2016161374A1 (en) 2015-04-03 2016-04-01 Oligonucleotide compounds for targeting huntingtin mrna
EP16716412.8A EP3277814B1 (en) 2015-04-03 2016-04-01 Oligonucleotide compounds for targeting huntingtin mrna

Publications (1)

Publication Number Publication Date
HRP20201200T1 true HRP20201200T1 (hr) 2020-11-13

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HRP20201200TT HRP20201200T1 (hr) 2015-04-03 2016-04-01 OLIGONUKLEOTIDNI SPOJEVI ZA CILJANJE HUNTINGTIN mRNK

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Country Link
US (5) US9809817B2 (https=)
EP (2) EP3766973A1 (https=)
JP (5) JP7009356B2 (https=)
CN (2) CN107980062B (https=)
AU (4) AU2016244027B2 (https=)
CA (1) CA2980337A1 (https=)
CY (1) CY1123289T1 (https=)
DK (1) DK3277814T3 (https=)
ES (1) ES2808750T3 (https=)
HR (1) HRP20201200T1 (https=)
HU (1) HUE050441T2 (https=)
LT (1) LT3277814T (https=)
PL (1) PL3277814T3 (https=)
PT (1) PT3277814T (https=)
RS (1) RS60762B1 (https=)
SI (1) SI3277814T1 (https=)
SM (1) SMT202000454T1 (https=)
WO (1) WO2016161374A1 (https=)

Families Citing this family (86)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2715289C (en) 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation Modified rnai polynucleotides and uses thereof
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
EP3560503B1 (en) 2010-03-24 2021-11-17 Phio Pharmaceuticals Corp. Rna interference in dermal and fibrotic indications
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
CA2879066C (en) 2012-07-13 2019-08-13 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
CN106061488B (zh) 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
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
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 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
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
US10900039B2 (en) 2014-09-05 2021-01-26 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
RS62672B1 (sr) 2015-04-03 2021-12-31 Univ Massachusetts Oligonukleotidna jedinjenja za tretman preeklampsije i drugih angiogenskih poremećaja
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
EP3862005A1 (en) 2015-07-06 2021-08-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
WO2017030973A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
US10478503B2 (en) * 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
KR102427379B1 (ko) * 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11299733B2 (en) 2016-07-15 2022-04-12 University Of Massachusetts Process of delivering small RNAs to sperm
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
ES2912270T3 (es) * 2016-11-23 2022-05-25 Mayo Found Medical Education & Res Transporte de productos biológicos por medio de partículas
CN110121558B (zh) * 2016-11-28 2023-06-13 日商那帕洁制药股份有限公司 化学修饰siRNA
DK3607069T3 (da) * 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
US12544344B2 (en) 2017-04-19 2026-02-10 Phio Pharmaceuticals Corp. Topical delivery of nucleic acid compounds
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
JP2020518259A (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． ハンチントン病治療組成物および方法
CA3065523A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP2020522510A (ja) * 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
JP7406793B2 (ja) * 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
EP3691657A4 (en) 2017-10-04 2021-07-21 Avidity Biosciences, Inc. NUCLEIC ACID POLYPEPTIDE COMPOSITIONS AND USES THEREOF
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3735252A4 (en) * 2018-01-04 2021-10-06 Avidity Biosciences, Inc. HETERODUPLEX NUCLEIC ACID MOLECULES AND USES THEREOF
CN111954548A (zh) * 2018-02-21 2020-11-17 亚克安娜生命科学有限公司 流体递送系统和方法
EP3769769A4 (en) * 2018-03-19 2022-05-04 National University Corporation Tokyo Medical and Dental University NUCLEIC ACID WITH REDUCED TOXICITY
SG11202009877XA (en) 2018-04-12 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
EP3781677A4 (en) 2018-04-16 2022-01-19 University of Massachusetts COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING
US12391942B2 (en) 2018-05-11 2025-08-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
CN113924364A (zh) * 2019-03-08 2022-01-11 罗格斯新泽西州立大学 治疗亨廷顿病的组合物和方法
US11820985B2 (en) * 2019-03-26 2023-11-21 University Of Massachusetts Modified oligonucleotides with increased stability
US11629347B2 (en) * 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
US12006499B2 (en) 2019-06-06 2024-06-11 Avidity Biosciences, Inc. Una amidites and uses thereof
CN114375296A (zh) 2019-06-06 2022-04-19 艾维迪提生物科学公司 核酸多肽组合物及其用途
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
US20220348912A1 (en) 2019-06-20 2022-11-03 University Of Massachusetts Compositions and methods for improved gene editing
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
EP4051795A1 (en) * 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
CN112980837B (zh) * 2019-12-13 2023-07-04 深圳艾码生物科技有限公司 一种抑制HTT基因表达的siRNA及其前体和应用
US20210363524A1 (en) * 2020-03-18 2021-11-25 University Of Massachusetts Oligonucleotides for snca modulation
JP7837560B2 (ja) 2020-03-26 2026-03-31 ユニバーシティー オブ マサチューセッツ 安定性の向上を有する修飾オリゴヌクレオチドの合成
US20210355491A1 (en) * 2020-04-20 2021-11-18 University Of Massachusetts Oligonucleotides for msh3 modulation
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
WO2022031591A2 (en) * 2020-08-03 2022-02-10 University Of Massachusetts Oligonucleotides for htt-1a modulation
US20240200063A1 (en) * 2021-03-24 2024-06-20 Atalanta Therapeutics, Inc. Microglial gene silencing using double-stranded sirna
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物
US20230340475A1 (en) * 2021-12-17 2023-10-26 University Of Massachusetts Oligonucleotides for mlh1 modulation
AU2023354001A1 (en) * 2022-09-28 2025-04-17 Atalanta Therapeutics, Inc. Compositions and methods for treatment of huntington's disease
IL325355A (en) * 2023-06-30 2026-02-01 Spark Therapeutics Inc RNA inhibitor targeting huntingtin expression
WO2025073871A1 (en) 2023-10-03 2025-04-10 Bicycletx Limited Bicyclic peptide ligand complexes specific for tfr1
WO2025160434A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease
WO2025265013A2 (en) * 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

Family Cites Families (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
ES2113371T3 (es) 1989-11-16 1998-05-01 Univ Duke Transformacion de celulas epidermicas de tejidos animales con la ayuda de particulas.
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) * 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
US5858988A (en) 1993-02-24 1999-01-12 Wang; Jui H. Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use
TW360548B (en) 1993-04-08 1999-06-11 Powderject Res Ltd Products for therapeutic use
WO1996033761A1 (en) 1995-04-28 1996-10-31 Medtronic, Inc. Intraparenchymal infusion catheter system
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US5735814A (en) 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6042820A (en) 1996-12-20 2000-03-28 Connaught Laboratories Limited Biodegradable copolymer containing α-hydroxy acid and α-amino acid units
US6472375B1 (en) 1998-04-16 2002-10-29 John Wayne Cancer Institute DNA vaccine and methods for its use
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
EP2447370B1 (en) 2001-09-28 2018-07-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
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)
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
KR101215701B1 (ko) 2002-07-19 2012-12-26 베스 이스라엘 데코니스 메디칼 센터 자간전증 또는 자간의 진단 및 치료 방법
AU2003291755A1 (en) 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
PT1633767T (pt) 2003-06-02 2019-02-27 Univ Massachusetts Métodos e composições para controlar a eficácia do silenciamento de arn
EP1633890B2 (en) 2003-06-02 2020-11-18 University of Massachusetts METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF FNAi
WO2008005562A2 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
EP1709195B1 (en) 2003-12-19 2014-01-22 Novartis Vaccines and Diagnostics, Inc. Cell transfecting formulations of small interfering rna, related compositions and methods of making and use
WO2005077333A2 (en) 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
US20060014289A1 (en) 2004-04-20 2006-01-19 Nastech Pharmaceutical Company Inc. Methods and compositions for enhancing delivery of double-stranded RNA or a double-stranded hybrid nucleic acid to regulate gene expression in mammalian cells
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
EP1937066A4 (en) 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES
JP5111385B2 (ja) * 2005-10-28 2013-01-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン遺伝子の発現を抑制するための組成物および方法
US20070259827A1 (en) 2006-01-25 2007-11-08 University Of Massachusetts Compositions and methods for enhancing discriminatory RNA interference
US9198981B2 (en) 2006-02-01 2015-12-01 The University Of Kentucky Modulation of angiogenesis
WO2007094218A1 (ja) 2006-02-16 2007-08-23 Gifu University 修飾オリゴヌクレオチド
US20090306178A1 (en) 2006-03-27 2009-12-10 Balkrishen Bhat Conjugated double strand compositions for use in gene modulation
US20080039415A1 (en) 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
WO2008154482A2 (en) 2007-06-08 2008-12-18 Sirnaomics, Inc. Sirna compositions and methods of use in treatment of ocular diseases
CN101199858A (zh) 2007-10-18 2008-06-18 广州拓谱基因技术有限公司 治疗眼科疾病的多靶点小干扰rna鸡尾酒制剂及制备方法
TW200927177A (en) 2007-10-24 2009-07-01 Nat Inst Of Advanced Ind Scien Lipid-modified double-stranded RNA having potent RNA interference effect
WO2009099991A2 (en) * 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2470656B1 (en) 2009-08-27 2015-05-06 Idera Pharmaceuticals, Inc. Composition for inhibiting gene expression and uses thereof
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
WO2012005898A2 (en) 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
CA2828544A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
US20140275211A1 (en) * 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
US20150299696A1 (en) 2012-05-02 2015-10-22 Sirna Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
KR101567576B1 (ko) 2012-05-22 2015-11-10 올릭스 주식회사 세포 내 관통능을 가지고 ｒｎａ 간섭을 유도하는 핵산 분자 및 그 용도
US9480752B2 (en) 2012-07-10 2016-11-01 Baseclick Gmbh Anandamide-modified nucleic acid molecules
US20150247141A1 (en) 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
US10513710B2 (en) * 2014-04-18 2019-12-24 University Of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
RS62672B1 (sr) 2015-04-03 2021-12-31 Univ Massachusetts Oligonukleotidna jedinjenja za tretman preeklampsije i drugih angiogenskih poremećaja
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides

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