JP2014512171A - コロイデレミアの遺伝子治療に使用するためのａａｖベクター - Google Patents

コロイデレミアの遺伝子治療に使用するためのａａｖベクター Download PDF

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JP2014512171A
JP2014512171A JP2013554938A JP2013554938A JP2014512171A JP 2014512171 A JP2014512171 A JP 2014512171A JP 2013554938 A JP2013554938 A JP 2013554938A JP 2013554938 A JP2013554938 A JP 2013554938A JP 2014512171 A JP2014512171 A JP 2014512171A
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vector
aav
rep1
sequence
seq
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JP2014512171A5 (cg-RX-API-DMAC7.html
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マクラーレン、ロバート
シーブラ、ミゲル
ジョン デュアリング、マシュウ
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ISISINNOVATION LIMITED
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ISISINNOVATION LIMITED
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2013554938A 2011-02-22 2012-02-21 コロイデレミアの遺伝子治療に使用するためのａａｖベクター Pending JP2014512171A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1103062.4A GB201103062D0 (en) 2011-02-22 2011-02-22 Method
GB1103062.4 2011-02-22
PCT/GB2012/050376 WO2012114090A1 (en) 2011-02-22 2012-02-21 Aav -vectors for use in gene therapy of choroideremia

Related Child Applications (1)

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JP2017117286A Division JP2017221192A (ja) 2011-02-22 2017-06-14 コロイデレミアの遺伝子治療に使用するためのａａｖベクター

Publications (2)

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JP2014512171A true JP2014512171A (ja) 2014-05-22
JP2014512171A5 JP2014512171A5 (cg-RX-API-DMAC7.html) 2015-04-16

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Family Applications (4)

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JP2013554938A Pending JP2014512171A (ja) 2011-02-22 2012-02-21 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2017117286A Pending JP2017221192A (ja) 2011-02-22 2017-06-14 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2019201092A Pending JP2020054345A (ja) 2011-02-22 2019-11-05 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2022193831A Pending JP2023036618A (ja) 2011-02-22 2022-12-02 コロイデレミアの遺伝子治療に使用するためのａａｖベクター

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JP2017117286A Pending JP2017221192A (ja) 2011-02-22 2017-06-14 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2019201092A Pending JP2020054345A (ja) 2011-02-22 2019-11-05 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2022193831A Pending JP2023036618A (ja) 2011-02-22 2022-12-02 コロイデレミアの遺伝子治療に使用するためのａａｖベクター

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US (4) US9834788B2 (cg-RX-API-DMAC7.html)
EP (2) EP2678435B2 (cg-RX-API-DMAC7.html)
JP (4) JP2014512171A (cg-RX-API-DMAC7.html)
KR (3) KR102299430B1 (cg-RX-API-DMAC7.html)
CN (2) CN103562396A (cg-RX-API-DMAC7.html)
AU (2) AU2012220404B2 (cg-RX-API-DMAC7.html)
BR (1) BR112013021318B1 (cg-RX-API-DMAC7.html)
CA (1) CA2827975C (cg-RX-API-DMAC7.html)
DK (1) DK2678435T3 (cg-RX-API-DMAC7.html)
ES (1) ES2676550T5 (cg-RX-API-DMAC7.html)
GB (1) GB201103062D0 (cg-RX-API-DMAC7.html)
HK (2) HK1258917A1 (cg-RX-API-DMAC7.html)
IL (1) IL228031B (cg-RX-API-DMAC7.html)
MX (2) MX388146B (cg-RX-API-DMAC7.html)
PL (1) PL2678435T3 (cg-RX-API-DMAC7.html)
PT (1) PT2678435T (cg-RX-API-DMAC7.html)
SG (1) SG192911A1 (cg-RX-API-DMAC7.html)
WO (1) WO2012114090A1 (cg-RX-API-DMAC7.html)

Cited By (5)

* Cited by examiner, † Cited by third party
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JP2018511652A (ja) * 2015-03-06 2018-04-26 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2019502378A (ja) * 2015-12-14 2019-01-31 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 眼疾患のための遺伝子療法
JP2019509762A (ja) * 2016-03-10 2019-04-11 ナイトスタアールエックス リミテッド プレニル化アッセイ
JP2021505619A (ja) * 2017-12-06 2021-02-18 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 発作障害の治療におけるｍｉｒ１０１またはｍｉｒ１２８の使用
JP2021520233A (ja) * 2018-04-05 2021-08-19 ナイトスター リミテッド Ａａｖ組成物、作製する方法及び使用の方法

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WO2015082690A1 (en) * 2013-12-06 2015-06-11 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
DK3218386T3 (da) 2014-11-14 2021-06-07 Voyager Therapeutics Inc Modulatorisk polynukleotid
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
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US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
JP2019534018A (ja) 2016-11-07 2019-11-28 スパーク セラピューティクス インコーポレイテッドＳｐａｒｋ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． Ｒａｂエスコートタンパク質の力価アッセイ
US11938197B2 (en) * 2017-01-10 2024-03-26 The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) Polynucleotides and vectors for the expression of transgenes
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
CN119491003A (zh) 2017-05-05 2025-02-21 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
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JP7766393B2 (ja) 2017-06-14 2025-11-10 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 眼疾患のための遺伝子療法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
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AU2018352236B2 (en) 2017-10-16 2025-04-10 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
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WO2019079488A1 (en) 2017-10-17 2019-04-25 Nightstarx Limited PRENYLATION ASSAY
PE20212076A1 (es) 2018-02-01 2021-10-26 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas
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JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
WO2019216932A1 (en) * 2018-05-07 2019-11-14 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
US12319929B2 (en) 2018-05-15 2025-06-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
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JP7585233B2 (ja) * 2019-04-30 2024-11-18 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 ポンペ病の治療のために有用な組成物
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
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Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018511652A (ja) * 2015-03-06 2018-04-26 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2021059561A (ja) * 2015-03-06 2021-04-15 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP7239550B2 (ja) 2015-03-06 2023-03-14 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2023071867A (ja) * 2015-03-06 2023-05-23 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2019502378A (ja) * 2015-12-14 2019-01-31 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 眼疾患のための遺伝子療法
JP7057281B2 (ja) 2015-12-14 2022-04-19 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 眼疾患のための遺伝子療法
JP2022088656A (ja) * 2015-12-14 2022-06-14 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 眼疾患のための遺伝子療法
JP2019509762A (ja) * 2016-03-10 2019-04-11 ナイトスタアールエックス リミテッド プレニル化アッセイ
JP7021122B2 (ja) 2016-03-10 2022-02-16 ナイトスタアールエックス リミテッド プレニル化アッセイ
JP2021505619A (ja) * 2017-12-06 2021-02-18 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 発作障害の治療におけるｍｉｒ１０１またはｍｉｒ１２８の使用
JP2021520233A (ja) * 2018-04-05 2021-08-19 ナイトスター リミテッド Ａａｖ組成物、作製する方法及び使用の方法

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