JP2009526067A5 - - Google Patents

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Publication number
JP2009526067A5
JP2009526067A5 JP2008554342A JP2008554342A JP2009526067A5 JP 2009526067 A5 JP2009526067 A5 JP 2009526067A5 JP 2008554342 A JP2008554342 A JP 2008554342A JP 2008554342 A JP2008554342 A JP 2008554342A JP 2009526067 A5 JP2009526067 A5 JP 2009526067A5
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JP
Japan
Prior art keywords
pharmaceutical composition
composition according
aav2
vector
administered
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JP2008554342A
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English (en)
Japanese (ja)
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JP2009526067A (ja
JP5706602B2 (ja
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Priority claimed from PCT/US2007/003388 external-priority patent/WO2007092563A2/en
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Publication of JP2009526067A5 publication Critical patent/JP2009526067A5/ja
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JP2008554342A 2006-02-08 2007-02-08 ニーマンピック病ａ型に対する遺伝子治療 Active JP5706602B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US77162806P 2006-02-08 2006-02-08
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2013092907A Division JP5881641B2 (ja) 2006-02-08 2013-04-25 ニーマンピック病ａ型に対する遺伝子治療

Publications (3)

Publication Number Publication Date
JP2009526067A JP2009526067A (ja) 2009-07-16
JP2009526067A5 true JP2009526067A5 (enExample) 2010-04-08
JP5706602B2 JP5706602B2 (ja) 2015-04-22

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ID=38345806

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2008554342A Active JP5706602B2 (ja) 2006-02-08 2007-02-08 ニーマンピック病ａ型に対する遺伝子治療
JP2013092907A Active JP5881641B2 (ja) 2006-02-08 2013-04-25 ニーマンピック病ａ型に対する遺伝子治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2013092907A Active JP5881641B2 (ja) 2006-02-08 2013-04-25 ニーマンピック病ａ型に対する遺伝子治療

Country Status (16)

Country Link
US (3) US20090117156A1 (enExample)
EP (2) EP1986661B1 (enExample)
JP (2) JP5706602B2 (enExample)
AR (1) AR059371A1 (enExample)
BR (1) BRPI0707590A2 (enExample)
CY (1) CY1121380T1 (enExample)
DK (1) DK1986661T3 (enExample)
ES (1) ES2700048T3 (enExample)
HU (1) HUE041928T2 (enExample)
IL (2) IL193165B (enExample)
LT (1) LT1986661T (enExample)
MX (3) MX360595B (enExample)
PL (1) PL1986661T3 (enExample)
PT (1) PT1986661T (enExample)
SI (1) SI1986661T1 (enExample)
WO (1) WO2007092563A2 (enExample)

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MX2007013734A (es) 2005-05-02 2008-03-14 Genzyme Corp Terapia genica para trastornos de la medula espinal.
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
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WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
KR20190057110A (ko) 2016-09-28 2019-05-27 코바, 인크. 치료적 mots-c 관련 펩타이드
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US11117930B2 (en) 2017-02-23 2021-09-14 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
CN110997693A (zh) 2017-06-07 2020-04-10 阿德克斯公司 τ聚集抑制剂
WO2018237066A1 (en) * 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services CODON OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF C1-TYPE NIEMANN-PICK DISEASE AND ASSOCIATED STATES
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
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
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
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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