MX2022002198A - Composiciones y métodos para modular el empalme y la expresión de proteínas. - Google Patents

Composiciones y métodos para modular el empalme y la expresión de proteínas.

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Publication number
MX2022002198A
MX2022002198A MX2022002198A MX2022002198A MX2022002198A MX 2022002198 A MX2022002198 A MX 2022002198A MX 2022002198 A MX2022002198 A MX 2022002198A MX 2022002198 A MX2022002198 A MX 2022002198A MX 2022002198 A MX2022002198 A MX 2022002198A
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Mexico
Prior art keywords
protein expression
methods
compositions
alternative splicing
splicing events
Prior art date
Application number
MX2022002198A
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English (en)
Inventor
Isabel Aznarez
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Stoke Therapeutics Inc
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Publication date
Application filed by Stoke Therapeutics Inc filed Critical Stoke Therapeutics Inc
Publication of MX2022002198A publication Critical patent/MX2022002198A/es

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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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Abstract

Los eventos de empalme alternativo pueden dar lugar a transcritos de ARNm no productivos que, a su vez, pueden conducir a la expresión aberrante de proteínas, y los agentes terapéuticos que pueden dirigirse a los eventos de empalme alternativo en los genes pueden modular el nivel de expresión de proteínas funcionales en pacientes enfermos y/o inhibir la expresión aberrante de proteínas. En el presente documento se describen agentes terapéuticos y métodos que pueden usarse para tratar una afección provocada por estos eventos de empalme alternativo.
MX2022002198A 2019-08-19 2020-08-19 Composiciones y métodos para modular el empalme y la expresión de proteínas. MX2022002198A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962888887P 2019-08-19 2019-08-19
US202063049262P 2020-07-08 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
MX2022002198A true MX2022002198A (es) 2022-05-24

Family

ID=74659533

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022002198A MX2022002198A (es) 2019-08-19 2020-08-19 Composiciones y métodos para modular el empalme y la expresión de proteínas.

Country Status (11)

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

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SG11202112946VA (en) * 2019-05-27 2021-12-30 Vision Pharma Pty Ltd Novel retinitis pigmentosa treatment
MX2022014155A (es) * 2020-05-11 2023-04-11 The Florey Inst Of Neuroscience And Mental Health Composiciones y metodos para tratar trastornos asociados a mutaciones de perdida de funcion en syngap1.
WO2022271699A2 (en) * 2021-06-21 2022-12-29 Stoke Therapeutics, Inc. Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
EP4373845A2 (en) * 2021-07-23 2024-05-29 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
WO2023196841A2 (en) * 2022-04-05 2023-10-12 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
WO2023220727A1 (en) * 2022-05-13 2023-11-16 The Trustees Of The University Of Pennsylvania Compositions for treating syngap-1 related neurodevelopmental disorders
WO2024005715A1 (en) * 2022-06-28 2024-01-04 Agency For Science, Technology And Research Oligonucleotides

Family Cites Families (13)

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EP2219680A2 (en) * 2007-11-13 2010-08-25 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
SG11201702682PA (en) * 2014-10-03 2017-04-27 Cold Spring Harbor Lab Targeted augmentation of nuclear gene output
KR20220105174A (ko) * 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
WO2017106292A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of kidney diseases
EP3389782A4 (en) * 2015-12-14 2019-07-31 Cold Spring Harbor Laboratory ANTISENSE OLIGOMERS FOR THE TREATMENT OF A POLYCYSTIC KIDNEY DISEASE
CA3005128A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
WO2017106283A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of liver diseases
JP7049248B2 (ja) * 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー
EP3390635A4 (en) * 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
WO2018098446A1 (en) * 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing
RS65031B1 (sr) * 2017-08-25 2024-02-29 Stoke Therapeutics Inc Antisens oligomeri za lečenje stanja i bolesti
CN111936163A (zh) * 2017-10-23 2020-11-13 斯托克制药公司 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体

Also Published As

Publication number Publication date
CN114746550A (zh) 2022-07-12
KR20220104677A (ko) 2022-07-26
JP2022544702A (ja) 2022-10-20
WO2021034985A1 (en) 2021-02-25
EP4017979A1 (en) 2022-06-29
AU2020334067A1 (en) 2022-03-17
CA3147970A1 (en) 2021-02-25
BR112022002905A2 (pt) 2022-07-12
US20220290142A1 (en) 2022-09-15
EP4017979A4 (en) 2024-03-27
IL290595A (en) 2022-04-01

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