JP7715640B2 - 異染性白質ジストロフィーの治療に有用な組成物 - Google Patents

異染性白質ジストロフィーの治療に有用な組成物

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JP7715640B2
JP7715640B2 JP2021564955A JP2021564955A JP7715640B2 JP 7715640 B2 JP7715640 B2 JP 7715640B2 JP 2021564955 A JP2021564955 A JP 2021564955A JP 2021564955 A JP2021564955 A JP 2021564955A JP 7715640 B2 JP7715640 B2 JP 7715640B2
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sequence
certain embodiments
seq
vector
harsa
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JP2022531861A5 (https=
JPWO2020227166A5 (https=
JP2022531861A (ja
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オルドー，ジュリエット
ウイルソン，ジェームス
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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
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    • A—HUMAN NECESSITIES
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    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
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    • C12N2750/14011—Parvoviridae
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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JP2021564955A 2019-05-03 2020-05-02 異染性白質ジストロフィーの治療に有用な組成物 Active JP7715640B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962843091P 2019-05-03 2019-05-03
US62/843,091 2019-05-03
PCT/US2020/031207 WO2020227166A1 (en) 2019-05-03 2020-05-02 Compositions useful in treatment of metachromatic leukodystrophy

Publications (4)

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JP2022531861A JP2022531861A (ja) 2022-07-12
JP2022531861A5 JP2022531861A5 (https=) 2023-05-12
JPWO2020227166A5 JPWO2020227166A5 (https=) 2023-05-12
JP7715640B2 true JP7715640B2 (ja) 2025-07-30

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US (2) US12338451B2 (https=)
EP (1) EP3962454A4 (https=)
JP (1) JP7715640B2 (https=)
KR (1) KR20220020261A (https=)
CN (1) CN114072129A (https=)
AR (1) AR118850A1 (https=)
AU (1) AU2020270421A1 (https=)
BR (1) BR112021021908A2 (https=)
CA (1) CA3135539A1 (https=)
CL (1) CL2021002881A1 (https=)
CO (1) CO2021016323A2 (https=)
IL (1) IL287715A (https=)
MX (1) MX2021013420A (https=)
PE (1) PE20220930A1 (https=)
SG (1) SG11202111867XA (https=)
TW (1) TW202108760A (https=)
WO (1) WO2020227166A1 (https=)

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EP3980447A4 (en) * 2019-06-10 2023-07-26 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR ARSA GENE TRANSFER AND METHODS OF USE THEREOF
US20230304034A1 (en) * 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
KR20230170022A (ko) * 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
RU2769577C1 (ru) * 2021-06-01 2022-04-04 федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) Препарат для лечения метахроматической лейкодистрофии и способ ее лечения
IL308787A (en) * 2021-06-08 2024-01-01 Univ Pennsylvania Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses
US20250177495A1 (en) * 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
WO2023196863A1 (en) * 2022-04-05 2023-10-12 Astellas Gene Therapies, Inc. Adeno-associated virus potency assay and uses thereof
AU2023273809A1 (en) * 2022-05-16 2025-01-02 Genzyme Corporation Methods of treating metachromatic leukodystrophy
WO2024036250A2 (en) * 2022-08-10 2024-02-15 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
WO2024079361A1 (en) * 2022-10-14 2024-04-18 Neuway Pharma Gmbh A PROTEIN- OR PEPTIDE-BASED CAPSULE (PPC), PREFERABLY A VLP, LOADED WITH A MESSENGER RNA (mRNA) AND A METHOD OF PRODUCTION AND PURIFICATION THEREOF
CN118389675B (zh) * 2024-05-27 2025-06-27 南方医科大学珠江医院 芳香族氨基酸脱羧酶作为标志物在肝性脑病的检测产品中的应用

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JP2013538788A (ja) 2010-06-25 2013-10-17 シャイアー ヒューマン ジェネティック セラピーズ， インコーポレイテッド アリールスルファターゼａのｃｎｓ送達の方法および組成物
JP2015509701A (ja) 2011-12-02 2015-04-02 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Ｃｎｓにおけるアリールスルファターゼａ活性を増加するための方法および組成物
JP2016523835A (ja) 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
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