JPWO2021034985A5 - - Google Patents

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Publication number
JPWO2021034985A5
JPWO2021034985A5 JP2022511203A JP2022511203A JPWO2021034985A5 JP WO2021034985 A5 JPWO2021034985 A5 JP WO2021034985A5 JP 2022511203 A JP2022511203 A JP 2022511203A JP 2022511203 A JP2022511203 A JP 2022511203A JP WO2021034985 A5 JPWO2021034985 A5 JP WO2021034985A5
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JP
Japan
Prior art keywords
fold
premrna
composition
target protein
exon
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
JP2022511203A
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English (en)
Japanese (ja)
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JP2022544702A5 (https=
JP2022544702A (ja
Publication date
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Priority claimed from PCT/US2020/047081 external-priority patent/WO2021034985A1/en
Publication of JP2022544702A publication Critical patent/JP2022544702A/ja
Publication of JPWO2021034985A5 publication Critical patent/JPWO2021034985A5/ja
Publication of JP2022544702A5 publication Critical patent/JP2022544702A5/ja
Pending legal-status Critical Current

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JP2022511203A 2019-08-19 2020-08-19 スプライシングおよびタンパク質発現を調節するための組成物および方法 Pending JP2022544702A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962888887P 2019-08-19 2019-08-19
US62/888,887 2019-08-19
US202063049262P 2020-07-08 2020-07-08
US63/049,262 2020-07-08
PCT/US2020/047081 WO2021034985A1 (en) 2019-08-19 2020-08-19 Compositions and methods for modulating splicing and protein expression

Publications (3)

Publication Number Publication Date
JP2022544702A JP2022544702A (ja) 2022-10-20
JPWO2021034985A5 true JPWO2021034985A5 (https=) 2023-08-28
JP2022544702A5 JP2022544702A5 (https=) 2023-08-28

Family

ID=74659533

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022511203A Pending JP2022544702A (ja) 2019-08-19 2020-08-19 スプライシングおよびタンパク質発現を調節するための組成物および方法

Country Status (11)

Country Link
US (1) US20220290142A1 (https=)
EP (1) EP4017979A4 (https=)
JP (1) JP2022544702A (https=)
KR (1) KR20220104677A (https=)
CN (1) CN114746550A (https=)
AU (1) AU2020334067A1 (https=)
BR (1) BR112022002905A2 (https=)
CA (1) CA3147970A1 (https=)
IL (1) IL290595A (https=)
MX (1) MX2022002198A (https=)
WO (1) WO2021034985A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020237294A1 (en) * 2019-05-27 2020-12-03 Murdoch University Novel retinitis pigmentosa treatment
EP4025345A4 (en) * 2019-09-03 2024-02-28 The Regents Of The University Of Colorado, A Body Corporate Systems, methods, and compositions for the rapid early-detection of host rna biomarkers of infection and early identification of covid-19 coronavirus infection in humans
AU2021272832A1 (en) * 2020-05-11 2022-12-15 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for treating disorders associated with loss-of-function mutations in SYNGAP1
EP4359538A4 (en) * 2021-06-21 2025-09-17 Stoke Therapeutics Inc ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DEGRADATION OF NONSENSE MRNA
WO2023004021A2 (en) * 2021-07-23 2023-01-26 The Children's Medical Center Corporation Zinc finger ccch-type containing 14 (zc3h14) mutants and methods of use
WO2023163801A1 (en) * 2022-02-24 2023-08-31 Q-State Biosciences, Inc. Therapeutics for syngap haploinsufficiency
US20250230467A1 (en) * 2022-04-05 2025-07-17 The Johns Hopkins University Methods and materials for treating syngap1-associated neurodevelopmental disorders
WO2023212625A1 (en) * 2022-04-28 2023-11-02 AcuraStem Incorporated Syf2 antisense oligonucleotides
WO2023220727A1 (en) * 2022-05-13 2023-11-16 The Trustees Of The University Of Pennsylvania Compositions for treating syngap-1 related neurodevelopmental disorders
CN119948160A (zh) * 2022-06-28 2025-05-06 新加坡科技研究局 寡核苷酸
WO2025090633A1 (en) * 2023-10-23 2025-05-01 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies
WO2025111249A2 (en) * 2023-11-20 2025-05-30 Dana-Farber Cancer Institute, Inc. Methods, kits and systems for determining sarcomatoid differentiation of renal cell carcinoma and methods for treating based on the same
GB202401412D0 (en) * 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009064920A2 (en) * 2007-11-13 2009-05-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
JP2012507989A (ja) * 2008-11-07 2012-04-05 センター ホスピタライヤー ユニヴェルシテール サント−ジュスティーヌ Syngap1機能不全ならびに精神遅滞の診断および治療用途でのその使用
CN107109411B (zh) * 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
EP3359685B1 (en) * 2015-10-09 2026-01-28 University Of Southampton Modulation of gene expression for deregulated protein expression
JP2018538287A (ja) * 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
WO2017106375A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of tuberous sclerosis complex
EP3390666A4 (en) * 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
CA3005090A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
EP3933041B1 (en) * 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
EP3390634A4 (en) * 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES
JP2019535789A (ja) * 2016-11-28 2019-12-12 ピーティーシー セラピューティクス,インコーポレーテッド Rnaスプライシングを調節する方法
SG11202001590RA (en) * 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
GB2610100B (en) * 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases

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