BRPI0707590A2 - terapia gÊnica para a doenÇa de niemann-pick do tipo a - Google Patents
terapia gÊnica para a doenÇa de niemann-pick do tipo a Download PDFInfo
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- BRPI0707590A2 BRPI0707590A2 BRPI0707590-1A BRPI0707590A BRPI0707590A2 BR PI0707590 A2 BRPI0707590 A2 BR PI0707590A2 BR PI0707590 A BRPI0707590 A BR PI0707590A BR PI0707590 A2 BRPI0707590 A2 BR PI0707590A2
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- brain
- aav2
- vector
- transgene
- mammalian
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| US77236006P | 2006-02-09 | 2006-02-09 | |
| US60/772,360 | 2006-02-09 | ||
| PCT/US2007/003388 WO2007092563A2 (en) | 2006-02-08 | 2007-02-08 | Gene therapy for niemann-pick disease type a |
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| BRPI0707590A2 true BRPI0707590A2 (pt) | 2011-05-10 |
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| PL2158322T3 (pl) * | 2007-06-06 | 2017-10-31 | Genzyme Corp | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| DK2561073T3 (en) * | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| US20130090374A1 (en) * | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
| JP2016523835A (ja) * | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| JP6879486B2 (ja) * | 2015-03-17 | 2021-06-02 | フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ | Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| EP3519431A1 (en) | 2016-09-28 | 2019-08-07 | Cohbar Inc. | Therapeutic mots-c related peptides |
| KR20190075964A (ko) | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 설계 |
| US20190269797A1 (en) * | 2016-11-04 | 2019-09-05 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
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| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| SG11202107761SA (en) | 2019-01-28 | 2021-08-30 | Cohbar Inc | Therapeutic peptides |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| SG11202110926XA (en) * | 2019-04-24 | 2021-11-29 | Univ Oregon Health & Science | Promoter sequence and related products and uses thereof |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| MX2022004353A (es) | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| JP2023532923A (ja) * | 2020-06-30 | 2023-08-01 | チルドレンズ ナショナル メディカル センター | 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用 |
| JP2023542614A (ja) | 2020-08-19 | 2023-10-11 | サレプタ セラピューティクス， インコーポレイテッド | レット症候群の治療のためのアデノ随伴ウイルスベクター |
| AU2022214529A1 (en) * | 2021-02-01 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of niemann pick type a disease |
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| AR059371A1 (es) | 2008-03-26 |
| PT1986661T (pt) | 2018-12-05 |
| WO2007092563A2 (en) | 2007-08-16 |
| PL1986661T3 (pl) | 2019-02-28 |
| US20090117156A1 (en) | 2009-05-07 |
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| EP3456331B1 (en) | 2021-05-26 |
| EP3456331A1 (en) | 2019-03-20 |
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| IL193165B (en) | 2018-04-30 |
| SI1986661T1 (sl) | 2018-12-31 |
| JP2013166777A (ja) | 2013-08-29 |
| JP5706602B2 (ja) | 2015-04-22 |
| US20230287366A1 (en) | 2023-09-14 |
| MX2018013684A (es) | 2021-06-15 |
| EP1986661A4 (en) | 2012-04-25 |
| ES2700048T3 (es) | 2019-02-13 |
| CY1121380T1 (el) | 2020-05-29 |
| MX360595B (es) | 2018-11-09 |
| EP1986661B1 (en) | 2018-08-29 |
| MX2018008355A (es) | 2021-06-15 |
| DK1986661T3 (da) | 2019-01-02 |
| WO2007092563A3 (en) | 2007-11-22 |
| JP2009526067A (ja) | 2009-07-16 |
| LT1986661T (lt) | 2018-12-10 |
| HUE041928T2 (hu) | 2019-06-28 |
| US20200080066A1 (en) | 2020-03-12 |
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