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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Applications Claiming Priority (5)
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| US60/771,628 | 2006-02-08 | ||
| 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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| BRPI0707590-1A BRPI0707590A2 (pt) | 2006-02-08 | 2007-02-08 | terapia gÊnica para a doenÇa de niemann-pick do tipo a |
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| PT3252161T (pt) * | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| EP3514232A1 (en) * | 2010-04-23 | 2019-07-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| WO2012145646A1 (en) * | 2011-04-20 | 2012-10-26 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis |
| RU2692251C2 (ru) * | 2013-05-15 | 2019-06-24 | Риджентс Оф Зэ Юниверсити Оф Миннесота | Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему |
| RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
| 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 |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
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| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| JP2019537576A (ja) * | 2016-11-04 | 2019-12-26 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
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| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
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| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CN113518784A (zh) | 2019-01-28 | 2021-10-19 | 科巴公司 | 治疗性肽 |
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| PH12021552661A1 (en) * | 2019-04-24 | 2023-02-27 | Univ Oregon Health & Science | Promoter sequence and related products and uses thereof |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| CN116209477A (zh) * | 2020-06-30 | 2023-06-02 | 儿童国家医疗中心 | 使用重组人酸性鞘氨醇酶改善骨骼肌纤维的修复 |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| CA3205351A1 (en) * | 2021-02-01 | 2022-08-04 | James M. Wilson | Compositions and methods for treatment of niemann pick type a disease |
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| JP5881641B2 (ja) | 2016-03-09 |
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| US20230287366A1 (en) | 2023-09-14 |
| MX360595B (es) | 2018-11-09 |
| PL1986661T3 (pl) | 2019-02-28 |
| JP5706602B2 (ja) | 2015-04-22 |
| IL258587A (en) | 2018-06-28 |
| US20090117156A1 (en) | 2009-05-07 |
| ES2700048T3 (es) | 2019-02-13 |
| EP1986661A4 (en) | 2012-04-25 |
| MX2018013684A (es) | 2021-06-15 |
| WO2007092563A3 (en) | 2007-11-22 |
| EP1986661B1 (en) | 2018-08-29 |
| SI1986661T1 (sl) | 2018-12-31 |
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