JP7436089B2 - 前頭側頭型認知症の治療 - Google Patents

前頭側頭型認知症の治療 Download PDF

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JP7436089B2
JP7436089B2 JP2018565253A JP2018565253A JP7436089B2 JP 7436089 B2 JP7436089 B2 JP 7436089B2 JP 2018565253 A JP2018565253 A JP 2018565253A JP 2018565253 A JP2018565253 A JP 2018565253A JP 7436089 B2 JP7436089 B2 JP 7436089B2
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JP2019511570A (ja
JP2019511570A5 (enExample
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プロトキン， アリス チェン
ディフネ アマード，
ジュリアン リーダース，
ビヴァリー エル． デビッドソン，
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Amado Defne
Chen Plotkin Alice
Reiders Julianne
Children's Hospital of Philadelphia
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Amado Defne
Chen Plotkin Alice
Reiders Julianne
Children's Hospital of Philadelphia
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    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • C—CHEMISTRY; METALLURGY
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    • 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

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  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (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)
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JP2018565253A 2016-03-02 2017-03-02 前頭側頭型認知症の治療 Active JP7436089B2 (ja)

Priority Applications (1)

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JP2022030769A JP7507808B2 (ja) 2016-03-02 2022-03-01 前頭側頭型認知症の治療

Applications Claiming Priority (3)

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US201662302525P 2016-03-02 2016-03-02
US62/302,525 2016-03-02
PCT/US2017/020397 WO2017151884A1 (en) 2016-03-02 2017-03-02 Therapy for frontotemporal dementia

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JP2019511570A5 JP2019511570A5 (enExample) 2020-03-19
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EP (1) EP3423109A4 (enExample)
JP (2) JP7436089B2 (enExample)
AU (1) AU2017227803B2 (enExample)
CA (1) CA3016314A1 (enExample)
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022523766A (ja) * 2019-02-22 2022-04-26 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 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
JP2022071067A (ja) * 2016-03-02 2022-05-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア 前頭側頭型認知症の治療

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EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US20240002459A1 (en) * 2016-07-14 2024-01-04 Emory University Granulin Compositions and Uses Related Thereto
CN117821509A (zh) 2017-04-03 2024-04-05 编码治疗公司 组织选择性转基因表达
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111542549A (zh) 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
MX2020004005A (es) 2017-10-03 2020-10-05 Prevail Therapeutics Inc Terapias genicas para los trastornos lisosomales.
EP3692158A4 (en) * 2017-10-03 2021-08-04 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
AU2018354195A1 (en) * 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CN118345115A (zh) * 2017-12-01 2024-07-16 编码治疗公司 工程化的dna结合蛋白
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US20210396772A1 (en) * 2018-10-16 2021-12-23 Denali Therapeutics Inc. Methods for treating and monitoring progranulin-associated disorders
EP3917539A4 (en) * 2019-02-01 2022-11-23 Avrobio, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROCOGNITIVE DISORDERS
JP7664848B2 (ja) 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
HRP20251506T1 (hr) 2019-04-10 2026-01-02 Prevail Therapeutics, Inc. Genska terapija za lizosomske poremećaje
SG11202113048SA (en) 2019-05-29 2021-12-30 Encoded Therapeutics Inc Compositions and methods for selective gene regulation
GB201913974D0 (en) 2019-09-27 2019-11-13 King S College London Vector
IL292382A (en) * 2019-10-22 2022-06-01 Applied Genetic Tech Corporation Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
EP4146797A1 (en) * 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
JP7820823B2 (ja) * 2020-05-27 2026-02-26 ウニヴェルジテート・チューリッヒ 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター
AU2021325891A1 (en) * 2020-08-10 2023-04-06 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
MX2023001701A (es) * 2020-08-12 2023-03-09 UCB Biopharma SRL Terapia genica utilizando constructos de acido nucleico que comprenden secuencias promotoras de la proteina de union a metil cpg 2 (mecp2).
AU2021332331A1 (en) * 2020-08-26 2023-03-09 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of GRN-associated adult-onset neurodegeneration
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration

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JP2009539847A (ja) 2006-06-07 2009-11-19 ジェンザイム・コーポレーション 筋萎縮性側索硬化症および他の脊髄障害のための遺伝子治療

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WO2009089635A1 (en) * 2008-01-16 2009-07-23 Neurodyn, Inc. Treating neurodegenerative diseases with progranulin (pgrn)
SI3988660T1 (sl) * 2009-05-02 2025-09-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
US20110203007A1 (en) * 2010-02-16 2011-08-18 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis
CA3016314A1 (en) 2016-03-02 2017-09-08 Julianne REIDERS Therapy for frontotemporal dementia

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JP2009539847A (ja) 2006-06-07 2009-11-19 ジェンザイム・コーポレーション 筋萎縮性側索硬化症および他の脊髄障害のための遺伝子治療

Non-Patent Citations (5)

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Title
Ｇｈｏｌｉｚａｄｅｈ， Ｓ． ｅｔ ａｌ．，Ｔｒａｎｓｄｕｃｔｉｏｎ ｏｆ ｔｈｅ ｃｅｎｔｒａｌ ｎｅｒｖｏｕｓ ｓｙｓｔｅｍ ａｆｔｅｒ ｉｎｔｒａｃｅｒｅｂｒｏｖｅｎｔｒｉｃｕｌａｒ ｉｎｊｅｃｔｉｏｎ ｏｆ ａｄｅｎｏ－ａｓｓｏｃｉａｔｅｄ ｖｉｒａｌ ｖｅｃｔｏｒｓ ｉｎ ｎｅｏｎａｔａｌ ａｎｄ ｊｕｖｅｎｉｌｅ ｍｉｃｅ，Ｈｕｍａｎ Ｇｅｎｅ Ｔｈｅｒａｐｙ Ｍｅｔｈｏｄｓ，２０１３年，Ｖｏｌ．２４， Ｎｏ．４，ｐ．２０５－２１３，ｄｏｉ：１０．１０８９／ｈｇｔｂ．２０１３．０７６
Ｋａｔｚ， Ｍ．Ｌ． ｅｔ ａｌ．，ＡＡＶ ｇｅｎｅ ｔｒａｎｓｆｅｒ ｄｅｌａｙｓ ｄｉｓｅａｓｅ ｏｎｓｅｔ ｉｎ ａ ＴＰＰ１－ｄｅｆｉｃｉｅｎｔ ｃａｎｉｎｅ ｍｏｄｅｌ ｏｆ ｔｈｅ ｌａｔｅ ｉｎｆａｎｔｉｌｅ ｆｏｒｍ ｏｆ Ｂａｔｔｅｎ ｄｉｓｅａｓｅ，Ｓｃｉｅｎｃｅ Ｔｒａｎｓｌａｔｉｏｎａｌ Ｍｅｄｉｃｉｎｅ，２０１５年，Ｖｏｌ．７， Ｎｏ．３１３，３１３ｒａ１８０，ｄｏｉ：１０．１１２６／ｓｃｉｔｒａｎｓｌｍｅｄ．ａａｃ６１９１
Ｍｉｎａｍｉ， Ｓ．Ｓ． ｅｔ ａｌ．，Ｐｒｏｇｒａｎｕｌｉｎ ｐｒｏｔｅｃｔｓ ａｇａｉｎｓｔ ａｍｙｌｏｉｄ β ｄｅｐｏｓｉｔｉｏｎ ａｎｄ ｔｏｘｉｃｉｔｙ ｉｎ Ａｌｚｈｅｉｍｅｒ’ｓ ｄｉｓｅａｓｅ ｍｏｕｓｅ ｍｏｄｅｌｓ，Ｎａｔｕｒｅ ｍｅｄｉｃｉｎｅ，２０１４年，Ｖｏｌ．２０， Ｎｏ．１０，ｐ．１１５７－１１６４，ｄｏｉ：１０．１０３８／ｎｍ．３６７２
Ｎｅｖｅｒｍａｎ， Ｎ．Ｊ． ｅｔ ａｌ．，Ｅｘｐｅｒｉｍｅｎｔａｌ ｔｈｅｒａｐｉｅｓ ｉｎ ｔｈｅ ｎｅｕｒｏｎａｌ ｃｅｒｏｉｄ ｌｉｐｏｆｕｓｃｉｎｏｓｅｓ，Ｂｉｏｃｈｉｍｉｃａ ｅｔ Ｂｉｏｐｈｙｓｉｃａ Ａｃｔａ，２０１５年，Ｖｏｌ．１８５２，ｐ．２２９２－２３００，ｄｏｉ：１０．１０１６／ｊ．ｂｂａｄｉｓ．２０１５．０４．０２６
Ｖａｎ Ｋａｍｐｅｎ Ｊ．Ｍ． ｅｔ ａｌ．，Ｐｒｏｇｒａｎｕｌｉｎ ｇｅｎｅ ｄｅｌｉｖｅｒｙ ｐｒｏｔｅｃｔｓ ｄｏｐａｍｉｎｅｒｇｉｃ ｎｅｕｒｏｎｓ ｉｎ ａ ｍｏｕｓｅ ｍｏｄｅｌ ｏｆ Ｐａｒｋｉｎｓｏｎ’ｓ ｄｉｓｅａｓｅ，ＰＬｏＳ Ｏｎｅ，２０１４年，Ｖｏｌ．９， Ｎｏ．５，ｅ９７０３２，ｄｏｉ：１０．１３７１／ｊｏｕｒｎａｌ．ｐｏｎｅ．００９７０３２

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022071067A (ja) * 2016-03-02 2022-05-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア 前頭側頭型認知症の治療
JP7507808B2 (ja) 2016-03-02 2024-06-28 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア 前頭側頭型認知症の治療
JP2022523766A (ja) * 2019-02-22 2022-04-26 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 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
JP7637058B2 (ja) 2019-02-22 2025-02-27 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 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス

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JP2019511570A (ja) 2019-04-25
JP2022071067A (ja) 2022-05-13
US20190328906A1 (en) 2019-10-31
EP3423109A4 (en) 2019-08-14
AU2017227803A1 (en) 2018-10-25
AU2017227803B2 (en) 2024-05-02
WO2017151884A1 (en) 2017-09-08
EP3423109A1 (en) 2019-01-09
CA3016314A1 (en) 2017-09-08

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