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
Original Assignee
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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    • C12N15/113Non-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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    • C12N15/113Non-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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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

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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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EP3976789A4 (en) * 2019-05-27 2023-08-09 Vision Pharma Pty Ltd NEW TREATMENT FOR RETINITIS PIGMENTOSA
JP2023526060A (ja) * 2020-05-11 2023-06-20 ザ フローリー インスティテュート オブ ニューロサイエンス アンド メンタル ヘルス Syngap1中の機能喪失変異に関連する障害を処置するための組成物及び方法
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
US20230265436A1 (en) * 2022-02-24 2023-08-24 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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WO2009064920A2 (en) * 2007-11-13 2009-05-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
CA2735129C (en) * 2008-11-07 2012-06-26 Centre Hospitalier Universitaire Sainte-Justine Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation
JP6867945B2 (ja) * 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強
WO2017060731A1 (en) * 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017106211A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of polycystic kidney disease
JP7049247B2 (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 腎臓病の処置のための組成物と方法
EP3933041B1 (en) * 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
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
CA3005128A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
MX2019005588A (es) * 2016-11-28 2019-10-15 Ptc Therapeutics Inc Metodos para modular corte y empalme de arn.
PL3673080T3 (pl) * 2017-08-25 2024-03-11 Stoke Therapeutics, Inc. Oligomery antysensowne do leczenia stanów i chorób
KR20200070367A (ko) * 2017-10-23 2020-06-17 스톡 테라퓨틱스, 인크. 넌센스 매개 rna 분해 기반 병태 및 질환 치료를 위한 안티센스 올리고머

Also Published As

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

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