CA3061655A1 - Gene therapy for neuronal ceroid lipofuscinoses - Google Patents
Gene therapy for neuronal ceroid lipofuscinoses Download PDFInfo
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- CA3061655A1 CA3061655A1 CA3061655A CA3061655A CA3061655A1 CA 3061655 A1 CA3061655 A1 CA 3061655A1 CA 3061655 A CA3061655 A CA 3061655A CA 3061655 A CA3061655 A CA 3061655A CA 3061655 A1 CA3061655 A1 CA 3061655A1
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- 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
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- C—CHEMISTRY; METALLURGY
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- C12Y—ENZYMES
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| US201862652006P | 2018-04-03 | 2018-04-03 | |
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| PCT/US2018/032278 WO2018209205A1 (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
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| EP3534892A4 (en) * | 2016-11-04 | 2020-05-27 | The Children's Hospital of Philadelphia | GENE TRANSFER COMPOSITIONS, METHODS AND USE FOR TREATING NEURODEGENERATIVE DISEASES |
| SG11202002457RA (en) | 2017-09-22 | 2020-04-29 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| CA3117982A1 (en) * | 2018-11-14 | 2020-05-22 | Bhargavi KONDRAGUNTA | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2020112802A1 (en) * | 2018-11-28 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| WO2020210324A1 (en) * | 2019-04-08 | 2020-10-15 | The Children's Hospital Of Philadelphia | Treatment of lysosomal storage disease in the eye through administration of aavs expressing tpp1 |
| CN114430684A (zh) * | 2019-04-12 | 2022-05-03 | 编码治疗公司 | 用于治疗剂投予的组成物及方法 |
| CN115803064A (zh) | 2020-05-12 | 2023-03-14 | 宾夕法尼亚州大学信托人 | 用于drg特异性降低转基因表达的组合物 |
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| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| ATE364718T1 (de) | 1997-04-01 | 2007-07-15 | Solexa Ltd | Verfahren zur vervielfältigung von nukleinsäure |
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| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
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| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| WO2002095073A1 (en) | 2001-05-18 | 2002-11-28 | Wisconsin Alumni Research Foundation | Method for the synthesis of dna sequences |
| SG168422A1 (en) | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
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| EP3409296A1 (en) | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
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| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
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| WO2010071832A1 (en) * | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
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| RU2018128780A (ru) * | 2013-07-26 | 2018-12-05 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
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| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
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| IL270422A (en) | 2020-01-30 |
| KR102719222B1 (ko) | 2024-10-21 |
| US20200239908A1 (en) | 2020-07-30 |
| BR112019023303A2 (pt) | 2020-06-16 |
| SG10201912401QA (en) | 2020-02-27 |
| IL270422B1 (en) | 2024-12-01 |
| WO2018209205A1 (en) | 2018-11-15 |
| WO2018209205A9 (en) | 2019-11-21 |
| US11591614B2 (en) | 2023-02-28 |
| RU2019139555A3 (enExample) | 2021-10-12 |
| RU2019139555A (ru) | 2021-06-11 |
| AU2018265531A1 (en) | 2019-11-14 |
| JP7273730B2 (ja) | 2023-05-15 |
| JP2023099113A (ja) | 2023-07-11 |
| US20230383312A1 (en) | 2023-11-30 |
| SG11201910015SA (en) | 2019-11-28 |
| JP2020519292A (ja) | 2020-07-02 |
| KR20200023280A (ko) | 2020-03-04 |
| EP3621612A4 (en) | 2021-06-16 |
| IL316926A (en) | 2025-01-01 |
| IL270422B2 (en) | 2025-04-01 |
| AU2018265531B2 (en) | 2024-07-11 |
| EP3621612A1 (en) | 2020-03-18 |
| US12428653B2 (en) | 2025-09-30 |
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