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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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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Priority to JP2022169285A priority Critical patent/JP2023002721A/ja
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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
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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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    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
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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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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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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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)
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US9818239B2 (en) 2015-08-20 2017-11-14 Zendrive, Inc. Method for smartphone-based accident detection
KR102760654B1 (ko) 2017-06-07 2025-02-04 리제너론 파마슈티칼스 인코포레이티드 효소 내재화를 위한 조성물 및 방법
US10559196B2 (en) 2017-10-20 2020-02-11 Zendrive, Inc. Method and system for vehicular-related communications
MA51796A (fr) 2018-02-07 2020-12-16 Regeneron Pharma Procédés et compositions pour l'administration de protéines thérapeutiques
US12252544B2 (en) 2018-05-17 2025-03-18 Regeneron Pharmaceuticals, Inc. Anti-CD63 antibodies, conjugates, and uses thereof
JP7624724B2 (ja) 2018-09-26 2025-01-31 カリフォルニア インスティテュート オブ テクノロジー 標的遺伝子療法のためのアデノ随伴ウイルス組成物
JP2022513034A (ja) * 2018-11-14 2022-02-07 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
AU2020215592A1 (en) * 2019-02-01 2021-08-12 Spark Therapeutics, Inc. AAV vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2
EP4042297A4 (en) 2019-12-03 2023-11-22 Zendrive, Inc. METHOD AND SYSTEM FOR DETERMINING THE RISK OF A ROUTE
WO2021180118A1 (en) * 2020-03-11 2021-09-16 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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RU2019117062A (ru) 2020-12-04
CN110198712A (zh) 2019-09-03
BR112019009074A2 (pt) 2019-07-16
MX2019005266A (es) 2019-09-27
AU2017355502B2 (en) 2023-08-31
AU2017355502A1 (en) 2019-05-16
JP2023002721A (ja) 2023-01-10
WO2018085688A1 (en) 2018-05-11
EP3534892A4 (en) 2020-05-27
RU2019117062A3 (enExample) 2021-03-11
US20190269797A1 (en) 2019-09-05
EP3534892A1 (en) 2019-09-11
CA3041548A1 (en) 2018-05-11

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