AU2018220212A1 - Gene therapy for treating familial hypercholesterolemia - Google Patents
Gene therapy for treating familial hypercholesterolemia Download PDFInfo
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- AU2018220212A1 AU2018220212A1 AU2018220212A AU2018220212A AU2018220212A1 AU 2018220212 A1 AU2018220212 A1 AU 2018220212A1 AU 2018220212 A AU2018220212 A AU 2018220212A AU 2018220212 A AU2018220212 A AU 2018220212A AU 2018220212 A1 AU2018220212 A1 AU 2018220212A1
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- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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- 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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- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C12N2830/00—Vector systems having a special element relevant for transcription
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| US62/461,015 | 2017-02-20 | ||
| PCT/US2018/018678 WO2018152485A1 (en) | 2017-02-20 | 2018-02-20 | Gene therapy for treating familial hypercholesterolemia |
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| AU2018220212A1 true AU2018220212A1 (en) | 2019-08-08 |
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| ES2876409T3 (es) | 2014-04-25 | 2021-11-12 | Univ Pennsylvania | Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol |
| IL303850A (en) | 2015-12-11 | 2023-08-01 | Univ Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| WO2021080975A1 (en) * | 2019-10-21 | 2021-04-29 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing cholesterol levels |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023081807A1 (en) * | 2021-11-04 | 2023-05-11 | The Trustees of the University of Pennsylvania Penn Center for Innovation | Compositions and methods for reducing pcsk9 levels in a subject |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| 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 |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | 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 |
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| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| EP1986737A1 (en) | 2006-02-24 | 2008-11-05 | Kenergy Inc | Class-e radio frequency amplifier for use with an implantable medical device |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| KR101010352B1 (ko) | 2008-05-30 | 2011-01-25 | 삼성중공업 주식회사 | 전력 제어 장치 및 방법 |
| BRPI1007155A2 (pt) | 2009-01-29 | 2017-05-30 | Univ Of California San Francisco | métodos para tratar um distúrbio neurológico cortical, e para dispensar um agente terapêutico para o córtex em um primata |
| US20120252877A1 (en) | 2009-08-14 | 2012-10-04 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| DK2529020T3 (en) | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2721063A4 (en) | 2011-06-20 | 2015-01-14 | Hoffmann La Roche | PCSK9 BINDING POLYPEPTIDES AND METHOD OF USE THEREOF |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| ES2992345T3 (es) * | 2011-09-16 | 2024-12-11 | Regeneron Pharma | Métodos para reducir los niveles de lipoproteína(a) mediante la administración de un inhibidor de la proproteína convertasa subtilisina kexina-9 (PCSK9) |
| AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| WO2014089212A1 (en) | 2012-12-05 | 2014-06-12 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of metabolic disorders |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| AU2014216160B2 (en) * | 2013-02-15 | 2017-03-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | AAV8 retinoschisin expression vector for treating X-linked retinoschisis |
| US9254332B2 (en) | 2013-03-15 | 2016-02-09 | Arecor Limited | Stable aqueous formulations of adenovirus vectors |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| JP2016538829A (ja) | 2013-10-03 | 2016-12-15 | モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 低密度リポタンパク質受容体をコードするポリヌクレオチド |
| ES2876409T3 (es) * | 2014-04-25 | 2021-11-12 | Univ Pennsylvania | Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| IL303850A (en) | 2015-12-11 | 2023-08-01 | Univ Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11554147B2 (en) * | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
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