KR20220104677A - 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법 - Google Patents

스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법 Download PDF

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KR20220104677A
KR20220104677A KR1020227008965A KR20227008965A KR20220104677A KR 20220104677 A KR20220104677 A KR 20220104677A KR 1020227008965 A KR1020227008965 A KR 1020227008965A KR 20227008965 A KR20227008965 A KR 20227008965A KR 20220104677 A KR20220104677 A KR 20220104677A
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premrna
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mrna
exon
nsae
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이사벨 아즈나레즈
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스톡 테라퓨틱스, 인크.
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    • C12N15/1138—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 against receptors or cell surface proteins
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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KR1020227008965A 2019-08-19 2020-08-19 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법 Pending KR20220104677A (ko)

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 (1)

Publication Number Publication Date
KR20220104677A true KR20220104677A (ko) 2022-07-26

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KR1020227008965A Pending KR20220104677A (ko) 2019-08-19 2020-08-19 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법

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

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JP7652713B2 (ja) * 2019-05-27 2025-03-27 ビジョン ファーマ ピーティーワイ リミテッド 新規の網膜色素変性処置
KR20220054401A (ko) * 2019-09-03 2022-05-02 더 리전츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트 감염의 숙주 rna 바이오마커의 신속한 조기-검출 및 인간의 covid-19 코로나바이러스 감염의 조기 식별을 위한 시스템, 방법 및 조성물
CN115916977A (zh) * 2020-05-11 2023-04-04 弗洛里神经科学与心理健康研究所 用于治疗与syngap1中功能失去突变相关的障碍的组合物和方法
CN117980479A (zh) * 2021-06-21 2024-05-03 斯托克制药公司 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体
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
EP4504951A2 (en) * 2022-04-05 2025-02-12 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
US20250313832A1 (en) * 2022-05-13 2025-10-09 The Trustees Of The University Of Pennsylvania Compositions for treating syngap-1 related neurodevelopmental disorders
US20250368991A1 (en) * 2022-06-28 2025-12-04 Agency For Science, Technology And Research Oligonucleotides
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

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WO2009064920A2 (en) * 2007-11-13 2009-05-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
EP2364325A4 (en) * 2008-11-07 2012-07-25 Ct Hospitalier Universitaire Sainte Justine SYNGAP1 DYSFUNCTIONS AND THEIR USE IN DIAGNOSTIC AND THERAPEUTIC APPLICATIONS FOR INTELLECTUAL SENTENCING
AU2015327836B2 (en) * 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CN108603230A (zh) * 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
WO2017106377A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
EP3390666A4 (en) * 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
EP3390635A4 (en) * 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
JP2019500345A (ja) * 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 肝臓病の処置のための組成物および方法
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
JP7036723B2 (ja) * 2015-12-14 2022-03-15 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
EA201991309A1 (ru) * 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
KR102318434B1 (ko) * 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
HUE070436T2 (hu) * 2017-10-23 2025-06-28 Stoke Therapeutics Inc Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére

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Publication number Publication date
IL290595A (en) 2022-04-01
MX2022002198A (es) 2022-05-24
US20220290142A1 (en) 2022-09-15
BR112022002905A2 (pt) 2022-07-12
JP2022544702A (ja) 2022-10-20
EP4017979A4 (en) 2024-03-27
CN114746550A (zh) 2022-07-12
AU2020334067A1 (en) 2022-03-17
CA3147970A1 (en) 2021-02-25
EP4017979A1 (en) 2022-06-29
WO2021034985A1 (en) 2021-02-25

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