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
Prior art date
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JP2008554342A
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English (en)
Japanese (ja)
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JP5706602B2 (ja
JP2009526067A (ja
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Priority claimed from PCT/US2007/003388 external-priority patent/WO2007092563A2/en
Publication of JP2009526067A publication Critical patent/JP2009526067A/ja
Publication of JP2009526067A5 publication Critical patent/JP2009526067A5/ja
Application granted granted Critical
Publication of JP5706602B2 publication Critical patent/JP5706602B2/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 (OSRAM) 2010-04-08
JP5706602B2 JP5706602B2 (ja) 2015-04-22

Family

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 (OSRAM)
EP (2) EP3456331B1 (OSRAM)
JP (2) JP5706602B2 (OSRAM)
AR (1) AR059371A1 (OSRAM)
BR (1) BRPI0707590A2 (OSRAM)
CY (1) CY1121380T1 (OSRAM)
DK (1) DK1986661T3 (OSRAM)
ES (1) ES2700048T3 (OSRAM)
HU (1) HUE041928T2 (OSRAM)
IL (2) IL193165B (OSRAM)
LT (1) LT1986661T (OSRAM)
MX (3) MX360595B (OSRAM)
PL (1) PL1986661T3 (OSRAM)
PT (1) PT1986661T (OSRAM)
SI (1) SI1986661T1 (OSRAM)
WO (1) WO2007092563A2 (OSRAM)

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ES2905616T3 (es) 2007-06-06 2022-04-11 Genzyme Corp Terapia génica para enfermedades de almacenamiento lisosómico
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9102949B2 (en) * 2010-04-23 2015-08-11 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
HK1222093A1 (zh) * 2013-05-15 2017-06-23 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
CA2964272A1 (en) 2014-10-21 2016-04-28 Guangping Gao Recombinant aav variants and uses thereof
EP3270944B1 (en) * 2015-03-17 2019-10-23 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
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SG11201808176TA (en) 2016-04-15 2018-10-30 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
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WO2018071831A1 (en) 2016-10-13 2018-04-19 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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WO2018226992A1 (en) 2017-06-07 2018-12-13 Adrx, Inc. Tau aggregation inhibitors
CA3068010A1 (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 niemann-pick type c1 deficiency and related conditions
CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
AU2020216135A1 (en) 2019-01-28 2021-08-05 Cohbar, Inc. Therapeutic peptides
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
US20220204988A1 (en) * 2019-04-24 2022-06-30 Oregon Health & Science University 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
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
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
JP2024506860A (ja) * 2021-02-01 2024-02-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ニーマンピック病ａ型を治療するための組成物及び方法

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