JP2019537576A - 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 - Google Patents

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 Download PDF

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JP2019537576A
JP2019537576A JP2019522377A JP2019522377A JP2019537576A JP 2019537576 A JP2019537576 A JP 2019537576A JP 2019522377 A JP2019522377 A JP 2019522377A JP 2019522377 A JP2019522377 A JP 2019522377A JP 2019537576 A JP2019537576 A JP 2019537576A
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aav
tpp1
particles
mammal
disease
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JP2019537576A5 (enExample
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ビバリー エル． デイビッドソン，
ビバリー エル． デイビッドソン，
ヨン ホン チェン，
ヨン ホン チェン，
ルイス テセドール，
ルイス テセドール，
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Children's Hospital of Philadelphia
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Children's Hospital of Philadelphia
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Priority to JP2022169285A priority Critical patent/JP2023002721A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
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    • A61K31/365—Lactones
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • 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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  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
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  • 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)
JP2019522377A 2016-11-04 2017-11-03 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 Pending JP2019537576A (ja)

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JP2022169285A JP2023002721A (ja) 2016-11-04 2022-10-21 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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US201662418033P 2016-11-04 2016-11-04
US62/418,033 2016-11-04
PCT/US2017/059986 WO2018085688A1 (en) 2016-11-04 2017-11-03 Gene transfer compositions, methods and uses for treating neurodegenerative diseases

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JP2019537576A true JP2019537576A (ja) 2019-12-26
JP2019537576A5 JP2019537576A5 (enExample) 2020-12-03

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JP2019522377A Pending JP2019537576A (ja) 2016-11-04 2017-11-03 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
JP2022169285A Pending JP2023002721A (ja) 2016-11-04 2022-10-21 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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US (1) US20190269797A1 (enExample)
EP (1) EP3534892A4 (enExample)
JP (2) JP2019537576A (enExample)
CN (1) CN110198712A (enExample)
AU (1) AU2017355502B2 (enExample)
BR (1) BR112019009074A2 (enExample)
CA (1) CA3041548A1 (enExample)
MX (1) MX2019005266A (enExample)
WO (1) WO2018085688A1 (enExample)

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US9818239B2 (en) 2015-08-20 2017-11-14 Zendrive, Inc. Method for smartphone-based accident detection
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
EP3698340A4 (en) 2017-10-20 2021-07-14 Zendrive, Inc. PROCEDURE AND SYSTEM FOR VEHICLE ASSOCIATED COMMUNICATION
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
KR20260086827A (ko) * 2018-11-14 2026-06-10 리젠엑스바이오 인크. 신경원성 세로이드 리포푸신증에 대한 유전자 요법
MX2021009250A (es) * 2019-02-01 2021-11-04 Spark Therapeutics Inc Metodos de tratamiento por vectores aav para la lipofuscinosis ceroidea neuronal infantil tardia tipo 2.
WO2021113475A1 (en) 2019-12-03 2021-06-10 Zendrive, Inc. Method and system for risk determination of a route
EP4118219A4 (en) * 2020-03-11 2024-04-17 Shanghai Belief-Delivery Biomed Co., Ltd. Novel use of aspirin compound in increasing nucleic acid expression
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

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US20150151007A1 (en) * 2007-06-06 2015-06-04 Genzyme Corporation Gene therapy for lysosomal storage diseases
JP2016530246A (ja) * 2013-07-26 2016-09-29 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 脳疾患を処置するための方法および組成物
WO2017070678A1 (en) * 2015-10-23 2017-04-27 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
JP2020519292A (ja) * 2017-05-11 2020-07-02 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 神経セロイドリポフスチン症のための遺伝子療法

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RU2019117062A3 (enExample) 2021-03-11
EP3534892A4 (en) 2020-05-27
CN110198712A (zh) 2019-09-03
JP2023002721A (ja) 2023-01-10
WO2018085688A1 (en) 2018-05-11
RU2019117062A (ru) 2020-12-04
AU2017355502A1 (en) 2019-05-16
EP3534892A1 (en) 2019-09-11
BR112019009074A2 (pt) 2019-07-16
US20190269797A1 (en) 2019-09-05
MX2019005266A (es) 2019-09-27
CA3041548A1 (en) 2018-05-11
AU2017355502B2 (en) 2023-08-31

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