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 PDFInfo
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- 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
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- A—HUMAN NECESSITIES
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- 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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- A—HUMAN NECESSITIES
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- 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
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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
- 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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- Animal Behavior & Ethology (AREA)
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- Biotechnology (AREA)
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- Gastroenterology & Hepatology (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
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| JP2022030769A JP7507808B2 (ja) | 2016-03-02 | 2022-03-01 | 前頭側頭型認知症の治療 |
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| JP2019511570A JP2019511570A (ja) | 2019-04-25 |
| JP2019511570A5 JP2019511570A5 (enExample) | 2020-03-19 |
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| JP2022030769A Active JP7507808B2 (ja) | 2016-03-02 | 2022-03-01 | 前頭側頭型認知症の治療 |
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| EP (1) | EP3423109A4 (enExample) |
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| AU (1) | AU2017227803B2 (enExample) |
| CA (1) | CA3016314A1 (enExample) |
| WO (1) | WO2017151884A1 (enExample) |
Cited By (2)
| 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 |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016073693A2 (en) | 2014-11-05 | 2016-05-12 | 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 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| AU2018346102B2 (en) | 2017-10-03 | 2023-05-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| JP7361037B2 (ja) * | 2017-10-03 | 2023-10-13 | プリベイル セラピューティクス，インコーポレーテッド | ライソゾーム病の遺伝子治療 |
| KR20200075865A (ko) * | 2017-10-23 | 2020-06-26 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환에 대한 유전자 요법 |
| CN118345115A (zh) * | 2017-12-01 | 2024-07-16 | 编码治疗公司 | 工程化的dna结合蛋白 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN113164627B (zh) * | 2018-10-16 | 2024-10-29 | 戴纳立制药公司 | 治疗和监测颗粒体蛋白前体相关病症的方法 |
| EP3917539A4 (en) * | 2019-02-01 | 2022-11-23 | Avrobio, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROCOGNITIVE DISORDERS |
| KR20210150486A (ko) | 2019-04-10 | 2021-12-10 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| MX2021012184A (es) | 2019-04-10 | 2022-01-24 | Prevail Therapeutics Inc | Terapias genicas para trastornos lisosomales. |
| GB201913974D0 (en) | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| AU2020371662A1 (en) * | 2019-10-22 | 2022-05-12 | Applied Genetic Technologies Corporation | Adeno-associated virus (AAV) systems for treatment of progranulin associated neurodegenerative diseases or disorders |
| US20230193212A1 (en) * | 2020-05-06 | 2023-06-22 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
| AU2021280943A1 (en) * | 2020-05-27 | 2022-07-28 | Universität Zürich | Viral vectors expressing therapeutic proteins specifically in myeloid cells and microglia |
| EP4192517A1 (en) * | 2020-08-10 | 2023-06-14 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| IL300294A (en) * | 2020-08-12 | 2023-04-01 | UCB Biopharma SRL | Gene therapy using nucleic acid constructs containing methyl CPG-binding protein 2 (MECP2) promoter sequences |
| WO2022046988A1 (en) * | 2020-08-26 | 2022-03-03 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| 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 |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
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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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| CA2712276A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| DK3421603T3 (da) * | 2009-05-02 | 2022-01-10 | Genzyme Corp | Genterapi for neurodegenerative forstyrrelser |
| 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 |
| JP7436089B2 (ja) * | 2016-03-02 | 2024-02-21 | 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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| Publication number | Priority date | Publication date | Assignee | Title |
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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)
| 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)
| 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 |
Also Published As
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| US20190328906A1 (en) | 2019-10-31 |
| EP3423109A1 (en) | 2019-01-09 |
| CA3016314A1 (en) | 2017-09-08 |
| EP3423109A4 (en) | 2019-08-14 |
| AU2017227803B2 (en) | 2024-05-02 |
| JP7507808B2 (ja) | 2024-06-28 |
| JP2019511570A (ja) | 2019-04-25 |
| JP2022071067A (ja) | 2022-05-13 |
| WO2017151884A1 (en) | 2017-09-08 |
| AU2017227803A1 (en) | 2018-10-25 |
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