MX2024010820A - Arnhc que se dirige a ubicaciones de snord115 para restaurar la expresión génica del ube3a paterno en el síndrome de angelman - Google Patents
Arnhc que se dirige a ubicaciones de snord115 para restaurar la expresión génica del ube3a paterno en el síndrome de angelmanInfo
- Publication number
- MX2024010820A MX2024010820A MX2024010820A MX2024010820A MX2024010820A MX 2024010820 A MX2024010820 A MX 2024010820A MX 2024010820 A MX2024010820 A MX 2024010820A MX 2024010820 A MX2024010820 A MX 2024010820A MX 2024010820 A MX2024010820 A MX 2024010820A
- Authority
- MX
- Mexico
- Prior art keywords
- snord115
- angelman syndrome
- restore
- locations
- gene expression
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N15/1137—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 enzymes
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/31—Combination therapy
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263317155P | 2022-03-07 | 2022-03-07 | |
| PCT/US2023/014680 WO2023172535A2 (en) | 2022-03-07 | 2023-03-07 | shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2024010820A true MX2024010820A (es) | 2024-12-06 |
Family
ID=87935834
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2024010820A MX2024010820A (es) | 2022-03-07 | 2024-09-04 | Arnhc que se dirige a ubicaciones de snord115 para restaurar la expresión génica del ube3a paterno en el síndrome de angelman |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20230332150A1 (https=) |
| EP (1) | EP4473116A2 (https=) |
| JP (1) | JP2025509262A (https=) |
| KR (1) | KR20240155345A (https=) |
| CN (1) | CN119384504A (https=) |
| AU (1) | AU2023231550A1 (https=) |
| CA (1) | CA3259398A1 (https=) |
| IL (1) | IL315359A (https=) |
| MX (1) | MX2024010820A (https=) |
| WO (1) | WO2023172535A2 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025101533A1 (en) * | 2023-11-09 | 2025-05-15 | Encoded Therapeutics, Inc. | Methods and compositions for increasing expression of ube3a |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20150329857A1 (en) * | 2011-12-28 | 2015-11-19 | Universite Pierre Et Marie Curie (Paris 6) | Rna interference to activate stem cells |
| PT3717646T (pt) * | 2017-12-01 | 2025-07-28 | Texas A & M Univ Sys | Tratamento antisense da síndrome de angelman |
| CA3113648A1 (en) * | 2018-09-21 | 2020-03-26 | University Of Connecticut | Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome |
| CA3133294A1 (en) * | 2019-03-15 | 2020-09-24 | Mir Scientific, Llc | Methods for predicting prostate cancer and uses thereof |
-
2023
- 2023-03-07 IL IL315359A patent/IL315359A/en unknown
- 2023-03-07 KR KR1020247033390A patent/KR20240155345A/ko active Pending
- 2023-03-07 CN CN202380026096.6A patent/CN119384504A/zh active Pending
- 2023-03-07 EP EP23767380.1A patent/EP4473116A2/en active Pending
- 2023-03-07 WO PCT/US2023/014680 patent/WO2023172535A2/en not_active Ceased
- 2023-03-07 US US18/179,716 patent/US20230332150A1/en active Pending
- 2023-03-07 AU AU2023231550A patent/AU2023231550A1/en active Pending
- 2023-03-07 JP JP2024553307A patent/JP2025509262A/ja active Pending
- 2023-03-07 CA CA3259398A patent/CA3259398A1/en active Pending
-
2024
- 2024-09-04 MX MX2024010820A patent/MX2024010820A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20240155345A (ko) | 2024-10-28 |
| IL315359A (en) | 2024-11-01 |
| CN119384504A (zh) | 2025-01-28 |
| JP2025509262A (ja) | 2025-04-11 |
| WO2023172535A2 (en) | 2023-09-14 |
| CA3259398A1 (en) | 2023-09-14 |
| AU2023231550A1 (en) | 2024-09-26 |
| EP4473116A2 (en) | 2024-12-11 |
| US20230332150A1 (en) | 2023-10-19 |
| WO2023172535A3 (en) | 2023-11-30 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| BR112023019229A2 (pt) | Composições e métodos para a liberação direcionada às células | |
| MX2021003455A (es) | Lípidos con amina ionizables. | |
| PH12021551279A1 (en) | Modified amine lipids | |
| MX2019001493A (es) | Agentes de iarn para infección causada por el virus de la hepatitis b. | |
| MX2023009704A (es) | Composiciones de arni de cetohexocinasa (khk) y métodos de uso de las mismas. | |
| CR20210572A (es) | Métodos y composiciones para editar ácido ribonucleicos (arn) | |
| CY1124066T1 (el) | 3' utr αλληλουχιες για τη σταθεροποιηση toy rna | |
| MX2021003303A (es) | Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano. | |
| CO2019014683A2 (es) | Vectores virales recombinantes con tropismo modificado y usos de estos para la introducción dirigida de material genético a células humanas | |
| WO2020051220A8 (en) | Compositions and methods for organ specific delivery of nucleic acids | |
| CO2017009083A2 (es) | Arni dirigido a la proteína hungtingtina, partículas víricas que lo contienen y métodos para reducir la toxicicidad del arni | |
| CO2018003678A2 (es) | Composiciones y métodos para inhibir la expresión génica de lpa | |
| PE20210921A1 (es) | Agentes arni como inhibidores de la transcripcion de la serpina 1 y composiciones que los comprenden | |
| MX2024010820A (es) | Arnhc que se dirige a ubicaciones de snord115 para restaurar la expresión génica del ube3a paterno en el síndrome de angelman | |
| EP4036079A3 (en) | Compounds and compositions for intracellular delivery of agents | |
| BR112017006469A2 (pt) | composições e métodos para inibição de expressão do gene hao1 (hidroxiácido oxidase 1 (glicolato oxidase)) | |
| BR112022017822A2 (pt) | Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas | |
| CL2025000194A1 (es) | Construcciones de arni para inhibir expresión de scap y sus usos | |
| MX2024015877A (es) | Estructuras lipídicas y composiciones que las comprenden | |
| MX2024010821A (es) | Arnhc dirigido a ube3a-ats para restaurar la expresión del gen ube3a paterno en el síndrome angelman | |
| WO2023081500A3 (en) | RNAi OLIGONUCLEOTIDE CONJUGATES | |
| CO2026003271A2 (es) | Composiciones de arni dirigidas contra el receptor de activina a tipo 1c (acvr1c) y métodos de uso de las mismas | |
| WO2024151745A3 (en) | Targeted anti-prdm compositions and uses thereof | |
| AR073800A1 (es) | Tratamiento de la infeccion del virus de la hepatitis c con sobreexpresion de micro rna -196 | |
| MX2022011487A (es) | Arnm transcrito in-vitro y composicion farmaceutica que comprende el mismo. |