KR102719222B1 - 신경 세로이드 지질갈색소증에 대한 유전자 요법 - Google Patents

신경 세로이드 지질갈색소증에 대한 유전자 요법 Download PDF

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KR102719222B1
KR102719222B1 KR1020197036041A KR20197036041A KR102719222B1 KR 102719222 B1 KR102719222 B1 KR 102719222B1 KR 1020197036041 A KR1020197036041 A KR 1020197036041A KR 20197036041 A KR20197036041 A KR 20197036041A KR 102719222 B1 KR102719222 B1 KR 102719222B1
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aav
tpp1
raav
mice
vector
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KR20200023280A (ko
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제임스 엠. 윌슨
네이선 카츠
크리스티안 힌더러
줄리엣 호르도
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
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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
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    • A61P25/00—Drugs for disorders of the nervous system
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    • 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
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    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485—Exopeptidases (3.4.11-3.4.19)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141—Use of virus, viral particle or viral elements as a vector
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    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020197036041A 2017-05-11 2018-05-11 신경 세로이드 지질갈색소증에 대한 유전자 요법 Active KR102719222B1 (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762504817P 2017-05-11 2017-05-11
US62/504,817 2017-05-11
US201762599816P 2017-12-18 2017-12-18
US62/599,816 2017-12-18
US201862652006P 2018-04-03 2018-04-03
US62/652,006 2018-04-03
PCT/US2018/032278 WO2018209205A1 (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Publications (2)

Publication Number Publication Date
KR20200023280A KR20200023280A (ko) 2020-03-04
KR102719222B1 true KR102719222B1 (ko) 2024-10-21

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US (2) US11591614B2 (enExample)
EP (1) EP3621612A4 (enExample)
JP (2) JP7273730B2 (enExample)
KR (1) KR102719222B1 (enExample)
AU (1) AU2018265531B2 (enExample)
BR (1) BR112019023303A2 (enExample)
CA (1) CA3061655A1 (enExample)
IL (2) IL316926A (enExample)
RU (1) RU2019139555A (enExample)
SG (2) SG11201910015SA (enExample)
WO (1) WO2018209205A1 (enExample)

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HK1222093A1 (zh) 2013-05-15 2017-06-23 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
JP2019537576A (ja) * 2016-11-04 2019-12-26 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ 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
CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
MX2021005517A (es) * 2018-11-14 2021-06-18 Regenxbio Inc Terapia genica para lipofuscinosis neuronal ceroidea.
CA3121211A1 (en) * 2018-11-28 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
JP2022527116A (ja) * 2019-04-08 2022-05-30 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア Ｔｐｐ１を発現するａａｖの投与による眼のリソソーム蓄積症の治療
JP2022526425A (ja) * 2019-04-12 2022-05-24 エンコーデッド セラピューティクス， インコーポレイテッド 治療剤の投与のための組成物および方法
KR20230010670A (ko) 2020-05-12 2023-01-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
JP2023537904A (ja) * 2020-08-10 2023-09-06 プリベイル セラピューティクス，インコーポレーテッド 神経変性障害のための遺伝子療法
CA3209779A1 (en) * 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
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JP7273730B2 (ja) 2023-05-15
IL270422B1 (en) 2024-12-01
JP2023099113A (ja) 2023-07-11
WO2018209205A1 (en) 2018-11-15
BR112019023303A2 (pt) 2020-06-16
AU2018265531B2 (en) 2024-07-11
US20230383312A1 (en) 2023-11-30
EP3621612A4 (en) 2021-06-16
EP3621612A1 (en) 2020-03-18
IL270422B2 (en) 2025-04-01
US20200239908A1 (en) 2020-07-30
US12428653B2 (en) 2025-09-30
RU2019139555A3 (enExample) 2021-10-12
IL270422A (en) 2020-01-30
SG11201910015SA (en) 2019-11-28
RU2019139555A (ru) 2021-06-11
WO2018209205A9 (en) 2019-11-21
CA3061655A1 (en) 2018-11-15
KR20200023280A (ko) 2020-03-04
JP2020519292A (ja) 2020-07-02
IL316926A (en) 2025-01-01
AU2018265531A1 (en) 2019-11-14
US11591614B2 (en) 2023-02-28
SG10201912401QA (en) 2020-02-27

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