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
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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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    • 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 JP2024045289A (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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JP2020503265A5 JP2020503265A5 (enExample) 2020-12-24
JPWO2018094251A5 JPWO2018094251A5 (enExample) 2022-04-13
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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 Pending JP2024045289A (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 Pending JP2024045289A (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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CA3044291A1 (en) * 2016-11-17 2018-05-24 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
CN120505370A (zh) 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
EP3801638A1 (en) 2018-06-08 2021-04-14 Novartis AG Cell-based assay for measuring drug product potency
TW202045530A (zh) * 2019-02-04 2020-12-16 美國全美兒童醫院之研究學會 腺相關病毒對ｃｌｎ３聚核苷酸的遞送
US20220389453A1 (en) * 2019-10-18 2022-12-08 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated with the irf2bpl gene
JP7684296B2 (ja) * 2019-11-22 2025-05-27 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 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法
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
WO2024235164A1 (zh) * 2023-05-15 2024-11-21 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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US20250312400A1 (en) 2025-10-09
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MX2019005874A (es) 2020-02-07
BR112019009834A2 (pt) 2019-10-08
AU2017362491A1 (en) 2019-06-06
CN118490845A (zh) 2024-08-16
EP3541429A1 (en) 2019-09-25
MA46863A (fr) 2019-09-25
RU2019118300A (ru) 2020-12-17
JP2024045289A (ja) 2024-04-02
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CN110325219A (zh) 2019-10-11
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US11583564B2 (en) 2023-02-21
CN110325219B (zh) 2024-03-26
SA519401806B1 (ar) 2023-03-08
IL266678A (en) 2019-07-31
US20200179467A1 (en) 2020-06-11
US12357663B2 (en) 2025-07-15
KR20190126047A (ko) 2019-11-08
AU2017362491B2 (en) 2023-02-02
JP2020503265A (ja) 2020-01-30
CL2019001354A1 (es) 2019-09-13
KR102641846B1 (ko) 2024-02-27
EP3541429A4 (en) 2020-08-12
RU2019118300A3 (enExample) 2021-01-11
US20230321164A1 (en) 2023-10-12
BR112019009834B1 (pt) 2022-05-17
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