KR102670852B1 - 변이형 RNAi - Google Patents

변이형 RNAi Download PDF

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KR102670852B1
KR102670852B1 KR1020177025286A KR20177025286A KR102670852B1 KR 102670852 B1 KR102670852 B1 KR 102670852B1 KR 1020177025286 A KR1020177025286 A KR 1020177025286A KR 20177025286 A KR20177025286 A KR 20177025286A KR 102670852 B1 KR102670852 B1 KR 102670852B1
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delete delete
vector
promoter
strand
nucleic acid
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KR20170110149A (ko
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리사 엠. 스타넥
아담 팔레르모
브렌다 리차드
세르지오 파블로 사르디
캐서린 오리어던
안토니우스 송
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젠자임 코포레이션
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KR1020177025286A 2015-02-10 2016-02-09 변이형 RNAi Active KR102670852B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
US62/114,578 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

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KR20170110149A KR20170110149A (ko) 2017-10-10
KR102670852B1 true KR102670852B1 (ko) 2024-05-29

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US (4) US10450563B2 (enExample)
EP (1) EP3256588A2 (enExample)
JP (2) JP2018506304A (enExample)
KR (1) KR102670852B1 (enExample)
CN (1) CN107438671B (enExample)
AR (1) AR103646A1 (enExample)
AU (3) AU2016219396B2 (enExample)
CA (1) CA3255657A1 (enExample)
CL (1) CL2017002027A1 (enExample)
CO (1) CO2017009083A2 (enExample)
CR (1) CR20170406A (enExample)
EA (1) EA201791805A1 (enExample)
EC (1) ECSP17059343A (enExample)
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IL (1) IL253893B (enExample)
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Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
IN2012DN00720A (enExample) 2009-07-06 2015-06-19 Ontorii Inc
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
MX347361B (es) 2011-07-19 2017-04-12 Wave Life Sciences Ltd Metodos para la sintesis de acidos nucleicos funcionalizados.
EP2872485B1 (en) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Asymmetric auxiliary group
EP2872147B1 (en) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Method for making chiral oligonucleotides
AU2013287630B2 (en) 2012-07-13 2017-05-25 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
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
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
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
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
MY181458A (en) 2015-02-10 2020-12-22 Genzyme Corp Variant rnai
JP6892433B2 (ja) 2015-04-03 2021-06-23 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 十分に安定化された非対称ｓｉｒｎａ
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JPWO2017068791A1 (ja) * 2015-10-23 2018-08-09 レナセラピューティクス株式会社 少なくとも１つのバルジ構造を有する核酸複合体
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
US10844377B2 (en) 2017-06-23 2020-11-24 University Of Massachusetts Two-tailed self-delivering siRNA
JP7248658B2 (ja) * 2017-09-22 2023-03-29 ジェンザイム・コーポレーション バリアントＲＮＡｉ
EP3684388A4 (en) * 2017-09-22 2021-09-22 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF HUNTINGTON'S DISEASE
AU2018378622A1 (en) * 2017-12-06 2020-05-28 Ovid Therapeutics Inc. Use of miR101 or miR128 in the treatment of seizure disorders
AU2019237514A1 (en) * 2018-03-23 2020-10-08 University Of Massachusetts Gene therapeutics for treating bone disorders
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
EP3826719A1 (en) 2018-07-24 2021-06-02 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
AU2019314529B2 (en) * 2018-08-03 2025-09-11 Genzyme Corporation Variant RNAi against alpha-synuclein
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
EP4461814A3 (en) * 2018-10-12 2025-02-26 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
CN113302302A (zh) * 2019-01-09 2021-08-24 科英布拉大学 双链rna及其用途
WO2020150636A1 (en) 2019-01-18 2020-07-23 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
US20220307024A1 (en) * 2019-06-17 2022-09-29 Alnylam Pharmaceuticals, Inc. Delivery of oligonucleotides to the striatum
MX2022001710A (es) 2019-08-09 2022-05-10 Univ Massachusetts Oligonucleótidos modificados químicamente dirigidos a los snp.
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021216556A2 (en) * 2020-04-20 2021-10-28 University Of Massachusetts Oligonucleotides for msh3 modulation
EP4442830A4 (en) 2021-11-29 2025-04-30 Shanghai Regenelead Therapies Co., Ltd. AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease
CN118660913A (zh) 2022-02-21 2024-09-17 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
WO2024187106A2 (en) * 2023-03-09 2024-09-12 Leal Therapeutics, Inc. Compositions and methods for modulating grin2a
WO2025178406A1 (ko) * 2024-02-21 2025-08-28 서울대학교산학협력단 Drosha 절단을 촉진시키는 구조적 모티프를 포함하는 rna 간섭 유도 핵산 분자의 변형 전구체

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009100502A1 (en) 2008-02-14 2009-08-20 Monash University Immunostimulatory sirna molecules
WO2011034811A1 (en) 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO2001011034A2 (en) 1999-08-09 2001-02-15 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
ATE513843T1 (de) * 2002-09-25 2011-07-15 Univ Massachusetts Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna
US7589189B2 (en) * 2003-05-14 2009-09-15 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US7902352B2 (en) * 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
CA2688514A1 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
CA2691617C (en) 2007-05-31 2020-07-21 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
EP2240783A2 (en) * 2008-02-04 2010-10-20 Galapagos N.V. Molecular targets and compounds, methods to identify the same, useful in the treatment of neurodegenerative diseases.
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
SG10201601110VA (en) 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
HK1203566A1 (zh) 2012-02-29 2015-10-30 桑格摩生物科学股份有限公司 治療亨廷頓氏病的方法和組合物
US9550989B2 (en) * 2013-10-03 2017-01-24 Washington University Rational design of microRNA-siRNA chimeras for multi-functional target suppression
MX376190B (es) 2014-03-21 2025-03-07 Genzyme Corp PARTÍCULAS DE rAAV QUE CODIFICAN miR-708 Y USOS DEL MISMO.
AU2015252797C1 (en) 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
KR102636633B1 (ko) 2014-12-24 2024-02-13 유니큐어 아이피 비.브이. 헌팅턴 유전자 억제를 유도하는 RNAi
MY181458A (en) 2015-02-10 2020-12-22 Genzyme Corp Variant rnai
JP7248658B2 (ja) 2017-09-22 2023-03-29 ジェンザイム・コーポレーション バリアントＲＮＡｉ

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009100502A1 (en) 2008-02-14 2009-08-20 Monash University Immunostimulatory sirna molecules
WO2011034811A1 (en) 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Cell,제151권,4호,900-911면(2012.11.9.) 1부.*

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