CN114746550A - 用于调节剪接和蛋白质表达的组合物和方法 - Google Patents

用于调节剪接和蛋白质表达的组合物和方法 Download PDF

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CN114746550A
CN114746550A CN202080073358.0A CN202080073358A CN114746550A CN 114746550 A CN114746550 A CN 114746550A CN 202080073358 A CN202080073358 A CN 202080073358A CN 114746550 A CN114746550 A CN 114746550A
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premrna
mrna
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nsae
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伊莎贝尔·阿兹纳雷兹
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Stoke Therapeutics Inc
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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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CN202080073358.0A 2019-08-19 2020-08-19 用于调节剪接和蛋白质表达的组合物和方法 Pending CN114746550A (zh)

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
CN114746550A true CN114746550A (zh) 2022-07-12

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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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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

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WO2016054615A2 (en) * 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CN109312343A (zh) * 2015-12-14 2019-02-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体

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WO2009064920A2 (en) * 2007-11-13 2009-05-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
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
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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

Patent Citations (3)

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WO2010051632A1 (en) * 2008-11-07 2010-05-14 Centre Hospitalier Universitaire Sainte-Justine Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation
WO2016054615A2 (en) * 2014-10-03 2016-04-07 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CN109312343A (zh) * 2015-12-14 2019-02-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体

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BR112022002905A2 (pt) 2022-07-12
WO2021034985A1 (en) 2021-02-25
KR20220104677A (ko) 2022-07-26
IL290595A (en) 2022-04-01
EP4017979A4 (en) 2024-03-27
EP4017979A1 (en) 2022-06-29
CA3147970A1 (en) 2021-02-25
AU2020334067A1 (en) 2022-03-17
US20220290142A1 (en) 2022-09-15
MX2022002198A (es) 2022-05-24
JP2022544702A (ja) 2022-10-20

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