CA3073937A1 - Recombinant adeno-associated vectors - Google Patents
Recombinant adeno-associated vectors Download PDFInfo
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- CA3073937A1 CA3073937A1 CA3073937A CA3073937A CA3073937A1 CA 3073937 A1 CA3073937 A1 CA 3073937A1 CA 3073937 A CA3073937 A CA 3073937A CA 3073937 A CA3073937 A CA 3073937A CA 3073937 A1 CA3073937 A1 CA 3073937A1
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- 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
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
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- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- 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
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| US201762550458P | 2017-08-25 | 2017-08-25 | |
| US62/550,458 | 2017-08-25 | ||
| PCT/US2018/047466 WO2019040586A1 (en) | 2017-08-25 | 2018-08-22 | RECOMBINANT ADENO-ASSOCIATE VECTORS |
Publications (1)
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| CA3073937A1 true CA3073937A1 (en) | 2019-02-28 |
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| JP2022546545A (ja) * | 2019-09-03 | 2022-11-04 | クリスタル バイオテック インコーポレイテッド | 先天性魚鱗癬の治療のための組成物及び方法 |
| IL298334A (en) | 2020-05-20 | 2023-01-01 | Certego Therapeutics Inc | Ring deuterated gaboxadol and its use for the treatment of psychiatric disorders |
| US20230330267A1 (en) * | 2020-09-04 | 2023-10-19 | Ohio State Innovation Foundation | Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain |
| WO2023044306A1 (en) * | 2021-09-14 | 2023-03-23 | California Institute Of Technology | Aav capsid variants |
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| ES2313784T3 (es) * | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| WO2000075365A2 (en) * | 1999-06-08 | 2000-12-14 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
| WO2004014222A2 (en) * | 2002-08-12 | 2004-02-19 | The Regents Of The University Of Michigan | Diagnosis and treatment of tuberous sclerosis |
| US7427396B2 (en) * | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| EP2357010B1 (en) * | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| EP2692868A1 (en) * | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| AU2013388083B2 (en) * | 2013-05-01 | 2019-08-22 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| HUE047996T2 (hu) * | 2013-07-22 | 2020-05-28 | Childrens Hospital Philadelphia | AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez |
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| WO2015171547A1 (en) * | 2014-05-05 | 2015-11-12 | Ovid Therapeutics Inc. | Methods of treating cognitive impairment associated with neurodegenerative disorders |
| US20160060655A1 (en) * | 2014-05-30 | 2016-03-03 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods to treat latent viral infections |
| WO2015191508A1 (en) * | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US10711270B2 (en) * | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| AU2016366549B2 (en) * | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| JP7082050B2 (ja) * | 2015-12-14 | 2022-06-07 | 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 | 脊髄性筋萎縮症の処置において有用なアデノ－関連ウイルスベクター |
| CN109476707B (zh) * | 2016-05-13 | 2022-12-02 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和其使用方法 |
| JP7291397B2 (ja) * | 2017-02-15 | 2023-06-15 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 血管系を通過する遺伝子移入の方法および組成物 |
| WO2018170223A1 (en) * | 2017-03-15 | 2018-09-20 | Ovid Therapeutics Inc. | Use of designer receptors exclusively activated by designer drugs in the treatment of seizure disorders |
| KR20200100735A (ko) * | 2017-12-20 | 2020-08-26 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 hM4Di의 용도 |
| CN112739353A (zh) * | 2018-06-14 | 2021-04-30 | 奥维德医疗公司 | Mir-92a或mir-145在治疗安格曼综合征中的用途 |
| KR20210084459A (ko) * | 2018-09-26 | 2021-07-07 | 캘리포니아 인스티튜트 오브 테크놀로지 | 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물 |
| US10557149B1 (en) * | 2019-07-15 | 2020-02-11 | Vigene Biosciences, Inc. | Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus |
| AU2020388634A1 (en) * | 2019-11-19 | 2022-06-23 | AskBio Inc. | Therapeutic adeno-associated virus comprising liver-specific promoters for treating Pompe disease and lysosomal disorders |
| WO2021113634A1 (en) * | 2019-12-05 | 2021-06-10 | The Board Of Regents Of The University Of Texas | Transgene cassettes designed to express a human mecp2 gene |
| CA3116391A1 (en) * | 2020-09-14 | 2022-03-14 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| KR20230152008A (ko) * | 2021-02-03 | 2023-11-02 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 단백질 m 유사체 및 융합 단백질 및 이들의 항체 기능 억제 용도 |
| US20240197919A1 (en) * | 2021-05-04 | 2024-06-20 | California Institute Of Technology | Recombinant aavs for delivery to central nervous system and brain vasculature |
| CN114259502A (zh) * | 2021-12-24 | 2022-04-01 | 南京鼓楼医院 | 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用 |
| CN115554418B (zh) * | 2022-11-22 | 2023-04-14 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
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| US20210371880A1 (en) | 2021-12-02 |
| WO2019040586A1 (en) | 2019-02-28 |
| MX2020002148A (es) | 2020-07-20 |
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| IL272655A (en) | 2020-03-31 |
| EP3655041A4 (en) | 2021-04-21 |
| JP2020533968A (ja) | 2020-11-26 |
| AU2018320849A1 (en) | 2020-03-05 |
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