JP7384797B2 - ムコ多糖症ｉｉｉｂ型のための遺伝子療法 - Google Patents
ムコ多糖症ｉｉｉｂ型のための遺伝子療法 Download PDFInfo
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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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- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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| US201762593090P | 2017-11-30 | 2017-11-30 | |
| US62/593,090 | 2017-11-30 | ||
| PCT/US2018/063166 WO2019108856A1 (en) | 2017-11-30 | 2018-11-29 | Gene therapy for mucopolysaccharidosis iiib |
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| JP2021507687A JP2021507687A (ja) | 2021-02-25 |
| JP2021507687A5 JP2021507687A5 (https=) | 2022-01-06 |
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| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| US12605465B2 (en) | 2020-03-23 | 2026-04-21 | The University Of North Carolina At Chapel Hill | AAV-NAGLU vectors for treatment of mucopolysaccharidosis IIIB |
| US20230183741A1 (en) * | 2020-05-11 | 2023-06-15 | University Of Florida Research Foundation, Incorporated | Disease correction by delivery of aav8 vectors expressing codon optimized naglu |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4288115A4 (en) * | 2021-02-08 | 2025-06-18 | Flagship Pioneering Innovations V, Inc. | Hybrid aav-anellovectors |
| WO2023077133A2 (en) * | 2021-10-29 | 2023-05-04 | The Johns Hopkins University | Controlling homeostatic regulatory circuitry in hypothalamus |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| WO2025157848A1 (en) * | 2024-01-23 | 2025-07-31 | Institut National de la Santé et de la Recherche Médicale | Methods for the intravenous treatment of sanfilippo syndrome type iiib |
Citations (3)
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| WO2016187017A1 (en) | 2015-05-15 | 2016-11-24 | Mcivor R Scott | Adeno-associated for therapeutic delivery to central nervous system |
| WO2017100682A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP2017523802A (ja) | 2014-05-14 | 2017-08-24 | ラボラトリオス デル ドクター エステヴェ ソシエダッド アノニマ | リソソーム蓄積症処置用のアデノ随伴ウイルスベクター |
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| US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2009131698A2 (en) * | 2008-04-23 | 2009-10-29 | Iowa State University Research Foundation, Inc. | PHOSPHORYLATED RECOMBINANT N-ACETYL-alpha-D- GLUCOSAMINIDASE (NaGlu) AND USES THEREOF |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| US8775146B2 (en) * | 2010-07-22 | 2014-07-08 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-N-acetylglucosaminidase |
| US20130039888A1 (en) * | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| WO2014125647A1 (ja) | 2013-02-18 | 2014-08-21 | 富士通オプティカルコンポーネンツ株式会社 | 光受信装置 |
| AU2014235096B2 (en) | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| US20190083583A1 (en) | 2016-01-29 | 2019-03-21 | Alexion Pharmaceuticals, Inc. | Enzyme replacement therapy in mucopolysaccharidosis iiib patients |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| WO2017136533A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
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2023
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Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2017523802A (ja) | 2014-05-14 | 2017-08-24 | ラボラトリオス デル ドクター エステヴェ ソシエダッド アノニマ | リソソーム蓄積症処置用のアデノ随伴ウイルスベクター |
| WO2016187017A1 (en) | 2015-05-15 | 2016-11-24 | Mcivor R Scott | Adeno-associated for therapeutic delivery to central nervous system |
| WO2017100682A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
Non-Patent Citations (1)
| Title |
|---|
| 北関東医学, 1959,(6):1342-1361 |
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| CN111989396A (zh) | 2020-11-24 |
| SG11202004991SA (en) | 2020-06-29 |
| US20200289675A1 (en) | 2020-09-17 |
| AU2018375163A1 (en) | 2020-06-11 |
| EP3717652A1 (en) | 2020-10-07 |
| JP2021507687A (ja) | 2021-02-25 |
| CL2020001428A1 (es) | 2020-10-23 |
| CA3083761A1 (en) | 2019-06-06 |
| KR20200104864A (ko) | 2020-09-04 |
| US11723989B2 (en) | 2023-08-15 |
| US20240009327A1 (en) | 2024-01-11 |
| IL274972A (en) | 2020-07-30 |
| WO2019108856A1 (en) | 2019-06-06 |
| BR112020010977A2 (pt) | 2020-11-17 |
| EA202091352A1 (ru) | 2020-08-27 |
| MX2020005673A (es) | 2020-12-03 |
| EP3717652A4 (en) | 2021-11-24 |
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