IL279391A - Use of MIR-92A or MIR-145 in the treatment of Engelmann syndrome - Google Patents
Use of MIR-92A or MIR-145 in the treatment of Engelmann syndromeInfo
- Publication number
- IL279391A IL279391A IL279391A IL27939120A IL279391A IL 279391 A IL279391 A IL 279391A IL 279391 A IL279391 A IL 279391A IL 27939120 A IL27939120 A IL 27939120A IL 279391 A IL279391 A IL 279391A
- Authority
- IL
- Israel
- Prior art keywords
- mir
- treatment
- angelman syndrome
- angelman
- syndrome
- Prior art date
Links
- 208000009575 Angelman syndrome Diseases 0.000 title 1
- 108091028684 Mir-145 Proteins 0.000 title 1
- 108091034121 miR-92a stem-loop Proteins 0.000 title 1
- 108091041519 miR-92a-3 stem-loop Proteins 0.000 title 1
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
-
- 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
-
- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K41/00—Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
- A61K41/0047—Sonopheresis, i.e. ultrasonically-enhanced transdermal delivery, electroporation of a pharmacologically active agent
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- 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
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862684774P | 2018-06-14 | 2018-06-14 | |
| PCT/US2019/037174 WO2019241624A1 (en) | 2018-06-14 | 2019-06-14 | Use of mir-92a or mir-145 in the treatment of angelman syndrome |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL279391A true IL279391A (en) | 2021-01-31 |
Family
ID=68839583
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL279391A IL279391A (en) | 2018-06-14 | 2020-12-13 | Use of MIR-92A or MIR-145 in the treatment of Engelmann syndrome |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US10704048B2 (https=) |
| EP (1) | EP3793567A4 (https=) |
| JP (1) | JP2021527087A (https=) |
| KR (1) | KR20210040358A (https=) |
| CN (1) | CN112739353A (https=) |
| AU (1) | AU2019285298A1 (https=) |
| CA (1) | CA3103267A1 (https=) |
| IL (1) | IL279391A (https=) |
| MX (1) | MX2020013667A (https=) |
| WO (1) | WO2019241624A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018320849A1 (en) * | 2017-08-25 | 2020-03-05 | Ovid Therapeutics Inc. | Recombinant adeno-associated vectors |
| KR20210040358A (ko) | 2018-06-14 | 2021-04-13 | 오비드 테라퓨틱스 인크. | 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 |
| WO2020227604A1 (en) * | 2019-05-08 | 2020-11-12 | Nova Southeastern University | Regulation of nucleotide excision repair (ner) by microrna for treatment of breast cancer |
| IL303239A (en) * | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5752515A (en) | 1996-08-21 | 1998-05-19 | Brigham & Women's Hospital | Methods and apparatus for image-guided ultrasound delivery of compounds through the blood-brain barrier |
| WO2006128245A1 (en) | 2005-06-03 | 2006-12-07 | Southern Adelaide Health Service-Flinders Medical Centre | Targeting cells with altered microrna expression |
| EP1937151A4 (en) | 2005-09-19 | 2011-07-06 | Univ Columbia | Systems and methods for opening of the blood-brain barrier of a subject using ultrasound |
| CN102676516B (zh) * | 2011-03-17 | 2014-06-04 | 中国医学科学院肿瘤研究所 | microRNA 145的新用途 |
| US20150267193A1 (en) * | 2011-10-14 | 2015-09-24 | Ramanuj Dasgupta | MicroRNAs and Methods of Using Same |
| EA201492116A1 (ru) * | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| WO2014004572A2 (en) | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| EP3291843B1 (en) | 2015-05-07 | 2023-03-22 | University of South Florida | Modified ube3a gene for a gene therapy approach for angelman syndrome |
| US20190298843A1 (en) * | 2015-05-11 | 2019-10-03 | Purdue Research Foundation | Ligand ionophore conjugates |
| EP4541420A3 (en) * | 2015-07-17 | 2025-06-25 | Ovid Therapeutics Inc. | Methods of treating developmental disorders with gaboxadol |
| KR20210040358A (ko) | 2018-06-14 | 2021-04-13 | 오비드 테라퓨틱스 인크. | 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 |
-
2019
- 2019-06-14 KR KR1020217001109A patent/KR20210040358A/ko not_active Withdrawn
- 2019-06-14 JP JP2020568984A patent/JP2021527087A/ja not_active Withdrawn
- 2019-06-14 WO PCT/US2019/037174 patent/WO2019241624A1/en not_active Ceased
- 2019-06-14 US US16/441,391 patent/US10704048B2/en active Active
- 2019-06-14 MX MX2020013667A patent/MX2020013667A/es unknown
- 2019-06-14 CN CN201980040006.2A patent/CN112739353A/zh active Pending
- 2019-06-14 EP EP19819943.2A patent/EP3793567A4/en not_active Withdrawn
- 2019-06-14 CA CA3103267A patent/CA3103267A1/en active Pending
- 2019-06-14 AU AU2019285298A patent/AU2019285298A1/en not_active Abandoned
-
2020
- 2020-07-07 US US16/922,273 patent/US11130952B2/en active Active
- 2020-12-13 IL IL279391A patent/IL279391A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2019241624A1 (en) | 2019-12-19 |
| JP2021527087A (ja) | 2021-10-11 |
| EP3793567A1 (en) | 2021-03-24 |
| MX2020013667A (es) | 2021-03-02 |
| US11130952B2 (en) | 2021-09-28 |
| CA3103267A1 (en) | 2019-12-19 |
| KR20210040358A (ko) | 2021-04-13 |
| EP3793567A4 (en) | 2021-08-11 |
| US20200332294A1 (en) | 2020-10-22 |
| CN112739353A (zh) | 2021-04-30 |
| US20190382761A1 (en) | 2019-12-19 |
| US10704048B2 (en) | 2020-07-07 |
| AU2019285298A1 (en) | 2021-01-07 |
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