JP7106534B2 - メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達 - Google Patents

メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達 Download PDF

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JP7106534B2
JP7106534B2 JP2019527321A JP2019527321A JP7106534B2 JP 7106534 B2 JP7106534 B2 JP 7106534B2 JP 2019527321 A JP2019527321 A JP 2019527321A JP 2019527321 A JP2019527321 A JP 2019527321A JP 7106534 B2 JP7106534 B2 JP 7106534B2
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mecp2
raav
avxs
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カスパール，ブライアン，ケー．
フォウスト，ケヴィン
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    • A61K35/76—Viruses; Subviral particles; Bacteriophages
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    • A—HUMAN NECESSITIES
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • 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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    • 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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JP2019527321A 2016-11-17 2017-11-17 メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達 Active JP7106534B2 (ja)

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JP2022112657A JP2022137210A (ja) 2016-11-17 2022-07-13 メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
JP2024008380A JP7862454B2 (ja) 2016-11-17 2024-01-24 メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達

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US201662423618P 2016-11-17 2016-11-17
US62/423,618 2016-11-17
PCT/US2017/062371 WO2018094251A1 (en) 2016-11-17 2017-11-17 Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2

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JP2022112657A Pending JP2022137210A (ja) 2016-11-17 2022-07-13 メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
JP2024008380A Active JP7862454B2 (ja) 2016-11-17 2024-01-24 メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達

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JP2024008380A Active JP7862454B2 (ja) 2016-11-17 2024-01-24 メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達

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Families Citing this family (8)

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IL322146A (en) * 2016-11-17 2025-09-01 Nationwide Childrens Hospital Inc Intrathecal administration of adenovirus encoding methyl-CpG-binding protein 2
EP4585229A3 (en) 2017-11-08 2026-01-14 Novartis AG Means and method for preparing viral vectors and uses of same
JP7478675B2 (ja) 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
AR118005A1 (es) * 2019-02-04 2021-09-08 Res Institute At Nationwide Children’S Hospital Administración del virus adenoasociado de polinucleótido de cln3
JP7781057B2 (ja) * 2019-10-18 2025-12-05 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
US20230211018A1 (en) * 2019-11-22 2023-07-06 Research Institute At Nationwide Children's Hospital Materials and methods for treatment of disorders associated with the ighmbp2 gene
WO2022221424A1 (en) 2021-04-13 2022-10-20 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus encoding methyl-cpg binding protein 2 for treating pitt hopkins syndrome via intrathecal delivery
CN121175427A (zh) * 2023-05-15 2025-12-19 上海金珂博生物技术有限公司 一种Rett综合症的基因治疗

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US12357663B2 (en) 2025-07-15
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BR112019009834B1 (pt) 2022-05-17
IL266678B2 (en) 2025-12-01
JP7862454B2 (ja) 2026-05-19
JP2020503265A (ja) 2020-01-30
AU2026200191A1 (en) 2026-01-29
CN110325219B (zh) 2024-03-26
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IL322146A (en) 2025-09-01
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MA46863A (fr) 2019-09-25
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RU2019118300A3 (enExample) 2021-01-11
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CA3044291A1 (en) 2018-05-24
BR112019009834A2 (pt) 2019-10-08
KR102641846B1 (ko) 2024-02-27
KR20190126047A (ko) 2019-11-08
CN110325219A (zh) 2019-10-11
SG10202105090WA (en) 2021-06-29
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