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CY1123289T1 - Ενωσεις ολιγονουκλεοτιδιων για τη στοχευση του huntingtin mrna - Google Patents

Ενωσεις ολιγονουκλεοτιδιων για τη στοχευση του huntingtin mrna

Info

Publication number
CY1123289T1
CY1123289T1 CY20201100791T CY201100791T CY1123289T1 CY 1123289 T1 CY1123289 T1 CY 1123289T1 CY 20201100791 T CY20201100791 T CY 20201100791T CY 201100791 T CY201100791 T CY 201100791T CY 1123289 T1 CY1123289 T1 CY 1123289T1
Authority
CY
Cyprus
Prior art keywords
huntingtin mrna
oligonucleotide compounds
targeting huntingtin
targeting
novel
Prior art date
Application number
CY20201100791T
Other languages
English (en)
Inventor
Anastasia Khvorova
Neil Aronin
Julia ALTERMAN
Original Assignee
University Of Massachusetts
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by University Of Massachusetts filed Critical University Of Massachusetts
Publication of CY1123289T1 publication Critical patent/CY1123289T1/el

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Abstract

Αυτή η γνωστοποίηση σχετίζεται με νέους στόχους huntingtin. Παρέχονται επίσης νέα ολιγονουκλεοτίδια για τη θεραπευτική αντιμετώπιση της νόσου του Huntington.
CY20201100791T 2015-04-03 2020-08-24 Ενωσεις ολιγονουκλεοτιδιων για τη στοχευση του huntingtin mrna CY1123289T1 (el)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
CY1123289T1 true CY1123289T1 (el) 2021-12-31

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CY20201100791T CY1123289T1 (el) 2015-04-03 2020-08-24 Ενωσεις ολιγονουκλεοτιδιων για τη στοχευση του huntingtin mrna

Country Status (18)

Country Link
US (5) US9809817B2 (el)
EP (2) EP3277814B1 (el)
JP (5) JP7009356B2 (el)
CN (2) CN116004624A (el)
AU (3) AU2016244027B2 (el)
CA (1) CA2980337A1 (el)
CY (1) CY1123289T1 (el)
DK (1) DK3277814T3 (el)
ES (1) ES2808750T3 (el)
HR (1) HRP20201200T1 (el)
HU (1) HUE050441T2 (el)
LT (1) LT3277814T (el)
PL (1) PL3277814T3 (el)
PT (1) PT3277814T (el)
RS (1) RS60762B1 (el)
SI (1) SI3277814T1 (el)
SM (1) SMT202000454T1 (el)
WO (1) WO2016161374A1 (el)

Families Citing this family (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009102427A2 (en) 2008-02-11 2009-08-20 Rxi Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
CA2753338A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Neutral nanotransporters
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
EP2370451B1 (en) 2008-12-02 2016-11-16 Wave Life Sciences Japan, Inc. Method for the synthesis of phosphorus atom modified nucleic acids
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
KR101885383B1 (ko) 2009-07-06 2018-08-03 웨이브 라이프 사이언시스 리미티드 신규한 핵산 프로드러그 및 그의 사용 방법
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
EP3578183B1 (en) 2010-03-24 2021-09-08 Phio Pharmaceuticals Corp. Rna interference in ocular indications
EP3560503B1 (en) 2010-03-24 2021-11-17 Phio Pharmaceuticals Corp. Rna interference in dermal and fibrotic indications
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 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
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
EP3079707A4 (en) 2013-12-02 2017-10-18 RXi Pharmaceuticals Corporation Immunotherapy of cancer
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
WO2015108048A1 (ja) 2014-01-15 2015-07-23 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
EP4137572A1 (en) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Chiral design
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
JP6836987B2 (ja) 2014-09-05 2021-03-03 フィオ ファーマシューティカルズ コーポレーションＰｈｉｏ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐ． Ｔｙｒまたはｍｍｐ１を標的とする核酸を用いて老化および皮膚障害を処置するための方法
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
CN107109407A (zh) 2014-11-14 2017-08-29 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
US9862952B2 (en) 2015-04-03 2018-01-09 University Of Massachusetts Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1（sod1）的核酸分子
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
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
CA3002744A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
CA3011894A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
AU2017268382B2 (en) * 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
KR102392236B1 (ko) 2016-05-18 2022-05-03 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2018013640A1 (en) 2016-07-15 2018-01-18 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
ES2903104T3 (es) * 2016-11-28 2022-03-31 Napajen Pharma Inc ARNip químicamente modificado
ES2932831T3 (es) * 2017-04-05 2023-01-26 Silence Therapeutics Gmbh Productos y composiciones
CA3061368A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP7557941B2 (ja) 2017-06-02 2024-09-30 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
EP3630199A4 (en) * 2017-06-02 2021-11-10 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE
EP3642341A4 (en) * 2017-06-23 2021-06-16 University Of Massachusetts Two-tailed self-delivering sirna and related methods
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症（ａｌｓ）の治療
EP3735252A4 (en) * 2018-01-04 2021-10-06 Avidity Biosciences, Inc. HETERODUPLEX NUCLEIC ACID MOLECULES AND THEIR USES
JP2021513903A (ja) * 2018-02-21 2021-06-03 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． 流体送達システムおよび方法
EP3769769A4 (en) * 2018-03-19 2022-05-04 National University Corporation Tokyo Medical and Dental University NUCLEIC ACID WITH REDUCED TOXICITY
WO2019204226A1 (en) 2018-04-16 2019-10-24 University Of Massachusetts Compositions and methods for improved gene editing
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando 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
EP3935167A2 (en) * 2019-03-08 2022-01-12 Rutgers, the State University of New Jersey Compositions and methods for treating huntington's disease
EP3946369A4 (en) * 2019-03-26 2023-10-18 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
US11578090B2 (en) 2019-06-06 2023-02-14 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
US20220307024A1 (en) * 2019-06-17 2022-09-29 Alnylam Pharmaceuticals, Inc. Delivery of oligonucleotides to the striatum
EP3987024A4 (en) 2019-06-20 2023-11-01 University Of Massachusetts COMPOSITIONS AND METHODS FOR ENHANCED GENOMIC EDITING
EP4010476A4 (en) 2019-08-09 2023-12-27 University Of Massachusetts Chemically modified oligonucleotides targeting snps
US20210108200A1 (en) * 2019-09-16 2021-04-15 University Of Massachusetts BRANCHED LIPID CONJUGATES OF siRNA FOR SPECIFIC TISSUE DELIVERY
CN114728018B (zh) * 2019-11-01 2024-07-19 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
CN115666659A (zh) 2020-03-26 2023-01-31 马萨诸塞大学 稳定性增加的修饰的寡核苷酸的合成
EP4138860A4 (en) * 2020-04-20 2024-08-07 University Of Massachusetts Oligonucleotides for msh3 modulation
US20220090069A1 (en) * 2020-08-03 2022-03-24 University Of Massachusetts Oligonucleotides for htt-1a modulation
JP2024512635A (ja) 2021-03-29 2024-03-19 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（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法
US11702659B2 (en) 2021-06-23 2023-07-18 University Of Massachusetts Optimized anti-FLT1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
US20230340475A1 (en) * 2021-12-17 2023-10-26 University Of Massachusetts Oligonucleotides for mlh1 modulation
WO2024073595A2 (en) * 2022-09-28 2024-04-04 Atalanta Therapeutics, Inc. Compositions and methods for treatment of huntington's disease

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
CA2068863C (en) 1989-11-16 2009-06-02 Stephen A. Johnston Particle-mediated transformation of animal tissue cells
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
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
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
TW404844B (en) 1993-04-08 2000-09-11 Oxford Biosciences Ltd Needleless syringe
AU5545596A (en) 1995-04-28 1996-11-18 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
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
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
JP4371812B2 (ja) 2001-09-28 2009-11-25 マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ マイクロｒｎａ分子
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)
WO2003092594A2 (en) 2002-04-30 2003-11-13 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
AP2005003230A0 (en) 2002-07-19 2005-03-31 Beth Israel Hospital Methods of diagnosing and treating pre-eclampsia or eclampsia.
WO2004044136A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2’-modified nucleosides for use in gene modulation
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
WO2004045543A2 (en) * 2002-11-14 2004-06-03 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
EP3502252B1 (en) 2003-06-02 2023-04-05 University of Massachusetts Methods and compositions for controlling efficacy of rna silencing
ES2357116T5 (es) 2003-06-02 2021-09-03 Univ Massachusetts Métodos y composiciones para mejorar la eficacia y especificad de FNAi
WO2008005562A2 (en) 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
US20060019912A1 (en) 2003-12-19 2006-01-26 Chiron Corporation Cell transfecting formulations of small interfering RNA related compositions and methods of making and use
US20060078542A1 (en) 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
AU2005222902B2 (en) 2004-03-12 2010-06-10 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
MXPA06012076A (es) 2004-04-20 2007-01-25 Nastech Pharm Co Metodos y composiciones para mejorar el suministro de arn bicatenario o un acido nucleico hibrido bicatenario para regular la expresion genetica en celulas de mamifero.
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
CA2568735A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
CA2619534A1 (en) * 2005-08-18 2007-02-22 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating neurological disease
AU2006305886C1 (en) * 2005-10-28 2011-03-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of huntingtin gene
WO2007087451A2 (en) 2006-01-25 2007-08-02 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
JPWO2007094218A1 (ja) 2006-02-16 2009-07-02 国立大学法人岐阜大学 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
US20110081362A1 (en) * 2008-01-31 2011-04-07 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
CA2753338A1 (en) * 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Neutral nanotransporters
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US8431544B1 (en) 2009-08-27 2013-04-30 Idera Pharmaceuticals, Inc. Compositions 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
EP2681315B1 (en) 2011-03-03 2017-05-03 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
WO2013176477A1 (ko) 2012-05-22 2013-11-28 비엠티 주식회사 세포 내 관통능을 가지는 rna 간섭을 유도하는 핵산 분자 및 그 용도
CA2877985C (en) 2012-07-10 2020-10-27 Baseclick Gmbh Anandamide-modified nucleic acid molecules
AU2013315225B2 (en) 2012-09-14 2018-11-08 Translate Bio Ma, Inc. Multimeric oligonucleotide compounds
WO2014076195A1 (en) 2012-11-15 2014-05-22 Santaris Pharma A/S Oligonucleotide conjugates
WO2015161184A1 (en) 2014-04-18 2015-10-22 University Of Massachusetts Exosomal loading using hydrophobically modified oligonucleotides
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
US9862952B2 (en) 2015-04-03 2018-01-09 University Of Massachusetts Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
CA3011894A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides

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