JP2023528435A5 - - Google Patents

Info

Publication number
JP2023528435A5
JP2023528435A5 JP2022574357A JP2022574357A JP2023528435A5 JP 2023528435 A5 JP2023528435 A5 JP 2023528435A5 JP 2022574357 A JP2022574357 A JP 2022574357A JP 2022574357 A JP2022574357 A JP 2022574357A JP 2023528435 A5 JP2023528435 A5 JP 2023528435A5
Authority
JP
Japan
Prior art keywords
oligonucleotide
spacer
bond
optionally
seq
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2022574357A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023528435A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/035603 external-priority patent/WO2021247800A2/en
Publication of JP2023528435A publication Critical patent/JP2023528435A/ja
Publication of JP2023528435A5 publication Critical patent/JP2023528435A5/ja
Pending legal-status Critical Current

Links

JP2022574357A 2020-06-03 2021-06-03 遺伝子転写物のモジュレーターを使用する神経学的疾患の処置 Pending JP2023528435A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063033926P 2020-06-03 2020-06-03
US63/033,926 2020-06-03
US202063119717P 2020-12-01 2020-12-01
US63/119,717 2020-12-01
PCT/US2021/035603 WO2021247800A2 (en) 2020-06-03 2021-06-03 Treatment of neurological diseases using modulators of gene transcripts

Publications (2)

Publication Number Publication Date
JP2023528435A JP2023528435A (ja) 2023-07-04
JP2023528435A5 true JP2023528435A5 (enExample) 2024-06-10

Family

ID=78831722

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022574357A Pending JP2023528435A (ja) 2020-06-03 2021-06-03 遺伝子転写物のモジュレーターを使用する神経学的疾患の処置

Country Status (8)

Country Link
US (1) US20230235332A1 (enExample)
EP (1) EP4162051A4 (enExample)
JP (1) JP2023528435A (enExample)
KR (1) KR20230043819A (enExample)
AU (1) AU2021284360A1 (enExample)
CA (1) CA3185488A1 (enExample)
IL (1) IL298647A (enExample)
WO (1) WO2021247800A2 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
WO2026080323A1 (en) 2024-10-09 2026-04-16 Quralis Corporation Treatment of neurological diseases using modulators of unc13a gene transcripts

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998013527A2 (en) * 1996-09-24 1998-04-02 Rapigene, Inc. Compositions and methods for enhancing hybridization specificity
US20110046200A1 (en) * 2004-08-03 2011-02-24 Michael T Howard Use of antisense oligonucleotides to effect translation modulation
CA2817256A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
CA3103429A1 (en) * 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
CA3126918A1 (en) * 2019-01-14 2020-07-23 President And Fellows Of Harvard College Methods and compositions for restoring stmn2 levels
EP3976010A4 (en) * 2019-06-03 2023-08-02 Quralis Corporation Oligonucleotides and methods of use for treating neurological diseases
EP4127172A4 (en) * 2020-03-25 2025-06-04 President and Fellows of Harvard College Methods and compositions for restoring STMN2 levels

Similar Documents

Publication Publication Date Title
JP2022536085A5 (enExample)
CN111373043B (zh) 用于降低snca表达的化合物和方法
KR100991975B1 (ko) 핵산을 기본으로 하는 화합물 및 이의 사용방법
JP2024543214A5 (enExample)
JP2013525330A5 (enExample)
JP2008510019A5 (enExample)
US20250051766A1 (en) Treatment of neurological diseases using modulators of unc13a gene transcripts
EP3765476B1 (en) Modified oligonucleotides for use in treatment of tauopathies
JP2023528435A5 (enExample)
JP2022520986A5 (enExample)
AU2022402929A1 (en) Splice switcher antisense oligonucleotides with modified backbone chemistries
IL313205A (en) Microbial consortia
JP2017510583A (ja) 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的unaオリゴマー
CN120505310A (zh) 用于调节smn2的化合物和方法
JP2025518487A5 (enExample)
US20230235332A1 (en) Treatment of neurological diseases using modulators of gene transcripts
US20230374519A1 (en) Compounds and methods for modulating pmp22
AU2024283557A1 (en) Modified unc13a oligonucleotides
CN119452086A (zh) 具有修饰的主链化学的gapmer反义寡核苷酸
JP2025501682A5 (enExample)
RU2021138237A (ru) Олигонуклеотиды и способы применения для лечения неврологических заболеваний
JP2023545041A (ja) Dmd患者におけるエクソン51スキッピングによるジストロフィンレスキューのためのオリゴマー化合物
JP2023532518A5 (enExample)
EA053024B1 (ru) Антисмысловые олигонуклеотиды-переключатели сплайсинга с модифицированной химией скелета
HK40121031A (zh) 具有经修饰的主链化学的剪接转换器反义寡核苷酸