BR112022002905A2 - Composições e métodos para modular splicing e expressão de proteína - Google Patents
Composições e métodos para modular splicing e expressão de proteínaInfo
- Publication number
- BR112022002905A2 BR112022002905A2 BR112022002905A BR112022002905A BR112022002905A2 BR 112022002905 A2 BR112022002905 A2 BR 112022002905A2 BR 112022002905 A BR112022002905 A BR 112022002905A BR 112022002905 A BR112022002905 A BR 112022002905A BR 112022002905 A2 BR112022002905 A2 BR 112022002905A2
- Authority
- BR
- Brazil
- Prior art keywords
- protein expression
- methods
- compositions
- modulate splicing
- alternative splicing
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Abstract
COMPOSIÇÕES E MÉTODOS PARA MODULAR SPLICING E EXPRESSÃO DE PROTEÍN. Eventos de splicing alternativos podem levar a transcritos de mRNA não produtivos que, por sua vez, podem levar à expressão aberrante de proteínas, e agentes terapêuticos que podem ter como alvo os eventos de splicing alternativos em genes podem modular o nível de expressão de proteínas funcionais em pacientes doentes e/ou inibir expressão de proteína aberrante. São descritos nesse documento agentes e métodos terapêuticos que podem ser usados para tratar uma condição causada por esses eventos de splicing alternativos.
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 |
---|---|
BR112022002905A2 true BR112022002905A2 (pt) | 2022-07-12 |
Family
ID=74659533
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022002905A BR112022002905A2 (pt) | 2019-08-19 | 2020-08-19 | Composições e métodos para modular splicing e expressão de proteína |
Country Status (11)
Country | Link |
---|---|
US (1) | US20220290142A1 (pt) |
EP (1) | EP4017979A4 (pt) |
JP (1) | JP2022544702A (pt) |
KR (1) | KR20220104677A (pt) |
CN (1) | CN114746550A (pt) |
AU (1) | AU2020334067A1 (pt) |
BR (1) | BR112022002905A2 (pt) |
CA (1) | CA3147970A1 (pt) |
IL (1) | IL290595A (pt) |
MX (1) | MX2022002198A (pt) |
WO (1) | WO2021034985A1 (pt) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
BR112021023936A2 (pt) * | 2019-05-27 | 2022-01-25 | Vision Pharma Pty Ltd | Tratamento inovador para retinite pigmentosa |
BR112022022893A2 (pt) * | 2020-05-11 | 2023-03-14 | The Florey Inst Of Neuroscience And Mental Health | Composições e métodos para tratamento de distúrbios associados a mutações de perda de função em syngap1 |
EP4359538A2 (en) * | 2021-06-21 | 2024-05-01 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases |
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 |
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)
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 |
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 |
EP3201339A4 (en) * | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
IL308174A (en) * | 2015-10-09 | 2024-01-01 | Univ Southampton | Gene expression modulation and dysregulated protein expression scanning |
EP3390634A4 (en) * | 2015-12-14 | 2019-08-14 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES |
EP3390635A4 (en) * | 2015-12-14 | 2019-05-01 | Cold Spring Harbor Laboratory | Antisense oligomers for the treatment of a tuberous sclerosis complex |
CA3005247A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of polycystic kidney disease |
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 |
WO2017106292A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of kidney diseases |
CA3043755A1 (en) * | 2016-11-28 | 2018-05-31 | Ptc Therapeutics, Inc. | Methods for modulating rna splicing |
KR20240035631A (ko) * | 2017-08-25 | 2024-03-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
EP3700570A4 (en) * | 2017-10-23 | 2021-08-04 | Stoke Therapeutics, Inc. | ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DECLINE OF NON-SENSE MEDIATION RNA |
-
2020
- 2020-08-19 CA CA3147970A patent/CA3147970A1/en active Pending
- 2020-08-19 JP JP2022511203A patent/JP2022544702A/ja active Pending
- 2020-08-19 WO PCT/US2020/047081 patent/WO2021034985A1/en unknown
- 2020-08-19 AU AU2020334067A patent/AU2020334067A1/en active Pending
- 2020-08-19 CN CN202080073358.0A patent/CN114746550A/zh active Pending
- 2020-08-19 BR BR112022002905A patent/BR112022002905A2/pt unknown
- 2020-08-19 KR KR1020227008965A patent/KR20220104677A/ko unknown
- 2020-08-19 EP EP20855637.3A patent/EP4017979A4/en active Pending
- 2020-08-19 MX MX2022002198A patent/MX2022002198A/es unknown
-
2022
- 2022-02-13 IL IL290595A patent/IL290595A/en unknown
- 2022-02-16 US US17/673,226 patent/US20220290142A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
MX2022002198A (es) | 2022-05-24 |
AU2020334067A1 (en) | 2022-03-17 |
IL290595A (en) | 2022-04-01 |
EP4017979A1 (en) | 2022-06-29 |
CN114746550A (zh) | 2022-07-12 |
KR20220104677A (ko) | 2022-07-26 |
JP2022544702A (ja) | 2022-10-20 |
CA3147970A1 (en) | 2021-02-25 |
US20220290142A1 (en) | 2022-09-15 |
EP4017979A4 (en) | 2024-03-27 |
WO2021034985A1 (en) | 2021-02-25 |
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