JP2023522701A - Ｍｓｈ３調節のためのオリゴヌクレオチド - Google Patents

Ｍｓｈ３調節のためのオリゴヌクレオチド Download PDF

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JP2023522701A
JP2023522701A JP2022563451A JP2022563451A JP2023522701A JP 2023522701 A JP2023522701 A JP 2023522701A JP 2022563451 A JP2022563451 A JP 2022563451A JP 2022563451 A JP2022563451 A JP 2022563451A JP 2023522701 A JP2023522701 A JP 2023522701A
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nucleotides
dsrna
antisense strand
strand
sense strand
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JP2023522701A5 (enExample
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コボロバ，アナスタシア
ファーガソン，シャンタル
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University of Massachusetts Amherst
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
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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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Wood Science & Technology (AREA)
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JP2022563451A 2020-04-20 2021-04-20 Ｍｓｈ３調節のためのオリゴヌクレオチド Pending JP2023522701A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063012603P 2020-04-20 2020-04-20
US63/012,603 2020-04-20
PCT/US2021/028166 WO2021216556A2 (en) 2020-04-20 2021-04-20 Oligonucleotides for msh3 modulation

Related Child Applications (1)

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JP2026063763A Division JP2026129735A (ja) 2020-04-20 2026-04-08 Ｍｓｈ３調節のためのオリゴヌクレオチド

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JP2023522701A true JP2023522701A (ja) 2023-05-31
JP2023522701A5 JP2023522701A5 (enExample) 2024-05-02

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JP2022563451A Pending JP2023522701A (ja) 2020-04-20 2021-04-20 Ｍｓｈ３調節のためのオリゴヌクレオチド

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US (1) US20210355491A1 (enExample)
EP (1) EP4138860A4 (enExample)
JP (1) JP2023522701A (enExample)
AU (1) AU2021260581A1 (enExample)
CA (1) CA3176204A1 (enExample)
WO (1) WO2021216556A2 (enExample)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3929293A3 (en) 2015-04-03 2022-03-16 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2018237245A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS
BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
WO2020041769A1 (en) 2018-08-23 2020-02-27 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
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
EP4146807A4 (en) * 2020-05-08 2024-09-04 Takeda Pharmaceuticals U.S.A., Inc. METHODS OF TREATING NUCLEOTIDE AMPLIFICATION DISEASES ASSOCIATED WITH MSH3 ACTIVITY
CA3174079A1 (en) 2020-05-26 2021-12-02 Anastasia Khvorova Synthetic oligonucleotides having regions of block and cluster modifications
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
EP4164656A4 (en) * 2020-06-11 2025-10-22 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING MSH3 EXPRESSION
BR112023019981A2 (pt) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
EP4423272A2 (en) * 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2023114989A2 (en) * 2021-12-17 2023-06-22 University Of Massachusetts Oligonucleotides for mlh3 modulation
US20240035027A1 (en) * 2022-05-04 2024-02-01 University Of Massachusetts Oligonucleotides for pms2 modulation
WO2023225495A2 (en) * 2022-05-16 2023-11-23 Atalanta Therapeutics, Inc. Compositions and methods for treatment of microsatellite dna expansion disorders
WO2024064954A2 (en) * 2022-09-23 2024-03-28 The Children's Hospital Of Philadelphia Compositions and methods for treating huntington's disease and related disorders
CN122341734A (zh) * 2023-10-11 2026-07-03 优尼科生物制药有限公司 重复扩增障碍中msh3的核酸调节

Citations (3)

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US20150307876A1 (en) * 2012-02-10 2015-10-29 Regents Of The University Of Minnesota Dna assimilation
US20170183655A1 (en) * 2014-05-07 2017-06-29 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
JP2019503394A (ja) * 2016-01-31 2019-02-07 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 分岐オリゴヌクレオチド

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AUPO974597A0 (en) * 1997-10-10 1997-11-06 Rhone-Poulenc Agro Methods for obtaining plant varieties
US7361468B2 (en) * 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
CA3255657A1 (en) * 2015-02-10 2025-12-01 Genzyme Corporation VARIANT RNAi
RS60762B1 (sr) * 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
US20170009304A1 (en) * 2015-07-07 2017-01-12 Splicingcodes.Com Method and kit for detecting fusion transcripts
WO2020117702A1 (en) * 2018-12-03 2020-06-11 Triplet Therapeutics, Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with msh3 activity

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150307876A1 (en) * 2012-02-10 2015-10-29 Regents Of The University Of Minnesota Dna assimilation
US20170183655A1 (en) * 2014-05-07 2017-06-29 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
JP2019503394A (ja) * 2016-01-31 2019-02-07 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 分岐オリゴヌクレオチド

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ANNE-MARIE M. GANNON ET AL., NUCLEIC ACIDS RESEARCH, vol. 40, no. 20, JPN6025012917, 2012, pages 10324 - 10333, ISSN: 0005748786 *
NIMRAT CHATTERJEE ET AL., DNA REPAIR, vol. 42, JPN6025012916, 2016, pages 26 - 32, ISSN: 0005748787 *

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Publication number Publication date
EP4138860A4 (en) 2024-08-07
WO2021216556A2 (en) 2021-10-28
AU2021260581A1 (en) 2022-11-24
CA3176204A1 (en) 2021-10-28
WO2021216556A3 (en) 2022-03-03
US20210355491A1 (en) 2021-11-18
EP4138860A2 (en) 2023-03-01

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