MA49923A - Système de déciblage de miarn pour interférence spécifique d'un tissu - Google Patents
Système de déciblage de miarn pour interférence spécifique d'un tissuInfo
- Publication number
- MA49923A MA49923A MA049923A MA49923A MA49923A MA 49923 A MA49923 A MA 49923A MA 049923 A MA049923 A MA 049923A MA 49923 A MA49923 A MA 49923A MA 49923 A MA49923 A MA 49923A
- Authority
- MA
- Morocco
- Prior art keywords
- miarn
- screening system
- specific interference
- fabric specific
- fabric
- Prior art date
Links
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- 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
- 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
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Dermatology (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762566966P | 2017-10-02 | 2017-10-02 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MA49923A true MA49923A (fr) | 2020-06-24 |
Family
ID=65994761
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA049923A MA49923A (fr) | 2017-10-02 | 2018-10-02 | Système de déciblage de miarn pour interférence spécifique d'un tissu |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20200248179A1 (fr) |
| EP (1) | EP3668997A4 (fr) |
| AU (1) | AU2018345772B2 (fr) |
| CA (1) | CA3077057A1 (fr) |
| MA (1) | MA49923A (fr) |
| WO (1) | WO2019070741A1 (fr) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3796919A4 (fr) * | 2018-05-15 | 2022-02-16 | Ramot at Tel-Aviv University Ltd. | Mir126-5p pour le traitement de maladies des motoneurones |
| AU2019316103B2 (en) | 2018-08-02 | 2026-02-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| GB201907882D0 (en) * | 2019-06-03 | 2019-07-17 | Cyprus Foundation For Muscular Dystrophy Res | Methods |
| AU2021237465A1 (en) | 2020-03-19 | 2022-10-13 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
| BR112022019889A2 (pt) | 2020-04-02 | 2023-03-07 | Mirecule Inc | Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente |
| US20230348906A1 (en) * | 2020-09-01 | 2023-11-02 | Ultragenyx Pharmaceutical Inc. | Dux4 inhibitors and methods of use thereof |
| TW202227627A (zh) | 2020-09-11 | 2022-07-16 | 美商愛羅海德製藥公司 | 用於抑制DUX4表現之RNAi藥劑、其組合物及使用方法 |
| CN116916938A (zh) * | 2020-12-31 | 2023-10-20 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途 |
| KR20230128314A (ko) * | 2020-12-31 | 2023-09-04 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을치료하기 위한 그의 용도 |
| CA3210662A1 (fr) * | 2021-02-03 | 2022-08-11 | Research Institute At Nationwide Children's Hospital | Compositions et methodes pour traiter une maladie associee a la surexpression de la dux4 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CN118265544A (zh) * | 2021-09-16 | 2024-06-28 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
| WO2023168202A2 (fr) * | 2022-03-02 | 2023-09-07 | Ultragenyx Pharmaceutical Inc. | Certains inhibiteurs dux4 et leurs procédés d'utilisation |
| WO2023196967A1 (fr) * | 2022-04-07 | 2023-10-12 | Kate Therapeutics, Inc. | Compositions et méthodes pour troubles musculaires |
| WO2025081023A1 (fr) * | 2023-10-11 | 2025-04-17 | Kate Therapeutics, Inc. | Compositions et méthodes pour traiter des troubles musculaires |
| WO2026011009A1 (fr) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions et procédés pour troubles musculaires |
| WO2026030242A1 (fr) | 2024-07-29 | 2026-02-05 | Kate Therapeutics, Inc. | Compositions de virus adéno-associés pour le traitement de la dystrophie musculaire facio-scapulo-humérale |
| CN118987019B (zh) * | 2024-08-30 | 2025-10-03 | 南华大学 | 基于miRNA的脂蛋白a调节方法及miR-653-5p的应用 |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| WO1995013392A1 (fr) | 1993-11-09 | 1995-05-18 | Medical College Of Ohio | Lignees cellulaires stables aptes a exprimer le gene de replication du virus adeno-associe |
| DE69433592T2 (de) | 1993-11-09 | 2005-02-10 | Targeted Genetics Corp., Seattle | Die erzielung hoher titer des rekombinanten aav-vektors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| EP0796339A1 (fr) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| CA2230758A1 (fr) | 1995-09-08 | 1997-03-13 | Genzyme Corporation | Vecteurs aav ameliores pour la therapie genique |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| CN1233291A (zh) | 1996-09-06 | 1999-10-27 | 宾西法尼亚大学托管会 | 重组腺伴随病毒定向基因治疗的方法 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| WO1999011764A2 (fr) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Procedes de generation de preparations de vecteurs de aav recombinants dont le titre est eleve et qui sont exemptes de virus assistant |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| WO2002053703A2 (fr) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Vecteurs aav2 et procedes |
| US7250289B2 (en) * | 2002-11-20 | 2007-07-31 | Affymetrix, Inc. | Methods of genetic analysis of mouse |
| WO2005013901A2 (fr) * | 2003-07-31 | 2005-02-17 | Isis Pharmaceuticals, Inc. | Composes oligomeres et compositions utilisables pour moduler des petits arn non-codants |
| US9217155B2 (en) * | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| WO2010138263A2 (fr) * | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Nouveaux virus adéno-associés (aav) et leurs utilisations |
| DK2826860T3 (en) * | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| EP2426203B1 (fr) | 2010-09-02 | 2016-07-13 | Université de Mons | Agents utiles pour le traitement de la dystrophie musculaire facio-scapulo-humérale |
| ES2739804T3 (es) * | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| CA2842798C (fr) * | 2011-07-25 | 2021-02-23 | Nationwide Children's Hospital, Inc. | Produits viraux recombinants et procedes pour inhibition de l'expression de dux4 |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| US9597380B2 (en) * | 2012-11-26 | 2017-03-21 | Modernatx, Inc. | Terminally modified RNA |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015141521A1 (fr) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | Appareil de traitement de substrat, procédé de fabrication de dispositif semi-conducteur et support d'enregistrement |
| WO2015164786A1 (fr) * | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Vecteurs de virus adéno-associés recombinants utiles pour réduire une immunité contre des produits transgéniques |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| SG11201804070XA (en) * | 2015-11-13 | 2018-06-28 | Baxalta Inc | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| KR102453187B1 (ko) * | 2016-04-02 | 2022-10-07 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 조직 특이적 발현을 위한 변형된 u6 프로모터 시스템 |
| US20190328791A1 (en) * | 2016-06-22 | 2019-10-31 | St. Jude Children's Research Hospital | Enhanced hetatopoietic stem cell transplantation |
| CN114127083B (zh) * | 2019-05-13 | 2024-11-15 | Dna2.0股份有限公司 | 使用人工微rna修饰哺乳动物细胞以改变其特性及其产品的组成 |
-
2018
- 2018-10-02 MA MA049923A patent/MA49923A/fr unknown
- 2018-10-02 WO PCT/US2018/054005 patent/WO2019070741A1/fr not_active Ceased
- 2018-10-02 US US16/651,814 patent/US20200248179A1/en active Pending
- 2018-10-02 EP EP18863924.9A patent/EP3668997A4/fr active Pending
- 2018-10-02 AU AU2018345772A patent/AU2018345772B2/en active Active
- 2018-10-02 CA CA3077057A patent/CA3077057A1/fr active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2018345772B2 (en) | 2025-04-17 |
| CA3077057A1 (fr) | 2019-04-11 |
| EP3668997A4 (fr) | 2021-05-19 |
| AU2018345772A1 (en) | 2020-04-23 |
| WO2019070741A1 (fr) | 2019-04-11 |
| US20200248179A1 (en) | 2020-08-06 |
| EP3668997A1 (fr) | 2020-06-24 |
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