JP5706602B2 - ニーマンピック病ａ型に対する遺伝子治療 - Google Patents

ニーマンピック病ａ型に対する遺伝子治療 Download PDF

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JP5706602B2
JP5706602B2 JP2008554342A JP2008554342A JP5706602B2 JP 5706602 B2 JP5706602 B2 JP 5706602B2 JP 2008554342 A JP2008554342 A JP 2008554342A JP 2008554342 A JP2008554342 A JP 2008554342A JP 5706602 B2 JP5706602 B2 JP 5706602B2
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aav2
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brain
transgene
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JP2009526067A5 (enExample
JP2009526067A (ja
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マルコ・エイ・パッシーニ
ロビン・ジェイ・ジーグラー
ジェイムズ・ドッジ
ラムヤ・シハブッディン
セン・エイチ・チェン
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Genzyme Corp
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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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)

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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

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

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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

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US (3) US20090117156A1 (enExample)
EP (2) EP3456331B1 (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)
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PL (1) PL1986661T3 (enExample)
PT (1) PT1986661T (enExample)
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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
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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
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IL258587A (en) 2018-06-28
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
JP5881641B2 (ja) 2016-03-09
EP3456331B1 (en) 2021-05-26
EP3456331A1 (en) 2019-03-20
EP1986661A2 (en) 2008-11-05
IL193165B (en) 2018-04-30
SI1986661T1 (sl) 2018-12-31
JP2013166777A (ja) 2013-08-29
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
BRPI0707590A2 (pt) 2011-05-10
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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