MX2022001984A - Terapia combinada transgénica y de miarn derivado de intrones para el tratamiento de ataxia espinocerebelosa 1 (sca1). - Google Patents
Terapia combinada transgénica y de miarn derivado de intrones para el tratamiento de ataxia espinocerebelosa 1 (sca1).Info
- Publication number
- MX2022001984A MX2022001984A MX2022001984A MX2022001984A MX2022001984A MX 2022001984 A MX2022001984 A MX 2022001984A MX 2022001984 A MX2022001984 A MX 2022001984A MX 2022001984 A MX2022001984 A MX 2022001984A MX 2022001984 A MX2022001984 A MX 2022001984A
- Authority
- MX
- Mexico
- Prior art keywords
- intron
- sca1
- treatment
- expression cassette
- derived mirna
- Prior art date
Links
- 108091070501 miRNA Proteins 0.000 title abstract 2
- 101100476779 Dictyostelium discoideum scaA gene Proteins 0.000 title 1
- 108700019146 Transgenes Proteins 0.000 title 1
- 101150053010 sca1 gene Proteins 0.000 title 1
- 238000002560 therapeutic procedure Methods 0.000 title 1
- 230000001225 therapeutic effect Effects 0.000 abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 108091010839 ATAXIN1-like Proteins 0.000 abstract 1
- 102100035029 Ataxin-1 Human genes 0.000 abstract 1
- 108010032963 Ataxin-1 Proteins 0.000 abstract 1
- 102100035021 Ataxin-1-like Human genes 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 239000002679 microRNA Substances 0.000 abstract 1
- 229940124788 therapeutic inhibitor Drugs 0.000 abstract 1
Classifications
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- 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
-
- 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
- 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]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Neurosurgery (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962887209P | 2019-08-15 | 2019-08-15 | |
| PCT/US2020/046499 WO2021030745A1 (en) | 2019-08-15 | 2020-08-14 | Combined transgene and intron-derived mirna therapy for treatment of sca1 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2022001984A true MX2022001984A (es) | 2022-05-19 |
Family
ID=74570777
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022001984A MX2022001984A (es) | 2019-08-15 | 2020-08-14 | Terapia combinada transgénica y de miarn derivado de intrones para el tratamiento de ataxia espinocerebelosa 1 (sca1). |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220288101A1 (OSRAM) |
| EP (1) | EP4013414A4 (OSRAM) |
| JP (1) | JP2022545378A (OSRAM) |
| CN (1) | CN114555084A (OSRAM) |
| AU (1) | AU2020330108A1 (OSRAM) |
| BR (1) | BR112022002794A2 (OSRAM) |
| CA (1) | CA3151122A1 (OSRAM) |
| MX (1) | MX2022001984A (OSRAM) |
| WO (1) | WO2021030745A1 (OSRAM) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11008627B2 (en) | 2019-08-15 | 2021-05-18 | Talis Biomedical Corporation | Diagnostic system |
| US20260043024A1 (en) * | 2022-08-26 | 2026-02-12 | The Children's Hospital Of Philadelphia | Mirnas targeting atnx2 for the treatment of als and sca2 |
| WO2024153769A1 (en) * | 2023-01-19 | 2024-07-25 | Uniqure Biopharma B.V. | Pharmaceutical formulations of gene delivery vehicles |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7001733B1 (en) * | 1998-05-12 | 2006-02-21 | Rigel Pharmaceuticals, Inc. | Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement |
| US20080274989A1 (en) * | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US7605249B2 (en) * | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2008150897A2 (en) * | 2007-05-31 | 2008-12-11 | University Of Iowa Research Foundation | Reduction of off-target rna interference toxicity |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| DK3628334T5 (da) * | 2014-03-21 | 2024-09-02 | Genzyme Corp | Genterapi til behandling af retinitis pigmentosa |
| ES2759317T3 (es) * | 2014-05-20 | 2020-05-08 | Univ Iowa Res Found | Compuestos terapéuticos para la enfermedad de Huntington |
| MA40782A (fr) * | 2014-10-03 | 2017-08-08 | Hocuslocus Llc | Procédés et compositions utilisant un arn non codant en vue de la culture et de la sélection de cellules |
| CA2977607A1 (en) * | 2015-02-26 | 2016-09-01 | Jacob Schneiderman | Methods and compositions relating to leptin antagonists |
| US11027024B2 (en) * | 2015-05-29 | 2021-06-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| US11118185B2 (en) * | 2016-03-01 | 2021-09-14 | University Of Florida Research Foundation, Incorporated | AAV vectors for treatment of dominant retinitis pigmentosa |
| CN111132699A (zh) * | 2017-09-08 | 2020-05-08 | 世代生物公司 | 修饰的封闭端dna(cedna) |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| KR20210042123A (ko) * | 2018-08-07 | 2021-04-16 | 더 칠드런스 호스피탈 오브 필라델피아 | 유전자 발현의 선택적 스플라이싱 조절 및 치료 방법 |
-
2020
- 2020-08-14 JP JP2022509153A patent/JP2022545378A/ja active Pending
- 2020-08-14 WO PCT/US2020/046499 patent/WO2021030745A1/en not_active Ceased
- 2020-08-14 BR BR112022002794A patent/BR112022002794A2/pt unknown
- 2020-08-14 US US17/632,587 patent/US20220288101A1/en active Pending
- 2020-08-14 EP EP20851939.7A patent/EP4013414A4/en active Pending
- 2020-08-14 MX MX2022001984A patent/MX2022001984A/es unknown
- 2020-08-14 AU AU2020330108A patent/AU2020330108A1/en active Pending
- 2020-08-14 CA CA3151122A patent/CA3151122A1/en active Pending
- 2020-08-14 CN CN202080072237.4A patent/CN114555084A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021030745A1 (en) | 2021-02-18 |
| CA3151122A1 (en) | 2021-02-18 |
| EP4013414A1 (en) | 2022-06-22 |
| JP2022545378A (ja) | 2022-10-27 |
| EP4013414A4 (en) | 2023-09-27 |
| BR112022002794A2 (pt) | 2022-08-09 |
| US20220288101A1 (en) | 2022-09-15 |
| AU2020330108A1 (en) | 2022-03-17 |
| CN114555084A (zh) | 2022-05-27 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EP4494706A3 (en) | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases | |
| WO2020198509A3 (en) | Modified oligonucleotides with increased stability | |
| MX2025001246A (es) | Compuestos de piperidina 3-sustituidos para inhibicion del protooncogen-b de linfoma de linea b de casitas (cbl-b) y uso de un inhibidor de cbl-b en combinacion con una vacuna contra el cancer y/o con un virus oncolitico | |
| MX2021013751A (es) | Compuestos de ciano ciclobutilo para la inhibicion del protooncogen b de linfoma de linaje b de casitas (cbl-b) y usos de los mismos. | |
| EP4170029A3 (en) | Modifying the specifity of plant non-coding rna molecules for silencing gene expression | |
| WO2006135436A3 (en) | Inhibition of gene expression and therapeutic uses thereof | |
| RU2016104161A (ru) | Ортогональные белки cas9 для рнк-направляемой регуляции и редактирования генов | |
| WO2019006418A3 (en) | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY | |
| MX2024002327A (es) | Sistemas y metodos para direccion con arn guia puntual (arngp) de adn endogeno y fuente. | |
| ZA201908445B (en) | Recombinant adenoviruses carrying transgenes | |
| MX2022002198A (es) | Composiciones y métodos para modular el empalme y la expresión de proteínas. | |
| MX2021004455A (es) | Composiciones y métodos para administrar transgenes. | |
| JOP20210142A1 (ar) | تركيبات rnai لتثبيط التعبير الوراثي عن pnpla3 وطرق استخدامها | |
| WO2009006453A8 (en) | Enabling the use of long dsrna for gene targeting in mammalian and other selected animal cells | |
| MX2022001080A (es) | Metodos y composiciones para reducir la inmunogenicidad mediante inhibidores de celulas b no agotadores. | |
| SG10201907746TA (en) | Methods of treating cancer harboring hemizygous loss of tp53 | |
| CO2023016031A2 (es) | Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos | |
| EP4335502A3 (en) | Modified u6 promoter system for tissue specific expression | |
| BR112022002794A2 (pt) | Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1 | |
| AU2019382824A8 (en) | Method and means to deliver miRNA to target cells | |
| SA520412423B1 (ar) | أوليجونيوكليوتيدات مضادة للحس تستهدف ألفا- سينيوكلين واستخداماتها | |
| ZA202103914B (en) | Cxcr7 inhibitors for the treatment of cancer | |
| Park et al. | Short hairpin RNA induces methylation of hepatitis B virus covalently closed circular DNA in human hepatoma cells | |
| WO2017087947A3 (en) | Method of treatment of follicular lymphoma with a btk inhibitor | |
| WO2020117840A3 (en) | Process to inhibit or eliminate eosinophilic diseases of the airway and related conditions |