KR102604159B1 - 조직 선택적 트랜스진 발현 - Google Patents

조직 선택적 트랜스진 발현 Download PDF

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KR102604159B1
KR102604159B1 KR1020197032432A KR20197032432A KR102604159B1 KR 102604159 B1 KR102604159 B1 KR 102604159B1 KR 1020197032432 A KR1020197032432 A KR 1020197032432A KR 20197032432 A KR20197032432 A KR 20197032432A KR 102604159 B1 KR102604159 B1 KR 102604159B1
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cases
transgene
expression
nucleic acid
selective
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KR20190137126A (ko
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스테파니 태글리아텔라
앤드류 영
슈-잉 첸
카틱 라마무티
데이비드 오버코플러
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엔코디드 테라퓨틱스, 인크.
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KR1020197032432A 2017-04-03 2018-04-03 조직 선택적 트랜스진 발현 Active KR102604159B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762480998P 2017-04-03 2017-04-03
US62/480,998 2017-04-03
PCT/US2018/025940 WO2018187363A1 (en) 2017-04-03 2018-04-03 Tissue selective transgene expression

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KR20190137126A KR20190137126A (ko) 2019-12-10
KR102604159B1 true KR102604159B1 (ko) 2023-11-20

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US (6) US10287607B2 (enExample)
EP (2) EP4403642A3 (enExample)
JP (2) JP7246359B2 (enExample)
KR (1) KR102604159B1 (enExample)
CN (2) CN117821509A (enExample)
AU (1) AU2018250161B2 (enExample)
BR (1) BR112019020777A2 (enExample)
CA (1) CA3058189A1 (enExample)
CL (2) CL2019002805A1 (enExample)
CO (1) CO2019011450A2 (enExample)
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AU2019358806B2 (en) 2018-10-08 2025-06-05 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
US20210395780A1 (en) * 2018-11-05 2021-12-23 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons
EP3921326A4 (en) * 2019-02-05 2022-12-14 The Broad Institute, Inc. SPECIFIC INTERNEURON THERAPEUTIC AGENTS TO NORMALIZE NEURON CELL EXCITABILITY AND TREAT DRAVET SYNDROME
AU2020228028A1 (en) 2019-02-25 2021-09-30 University Of Massachusetts DNA-binding domain transactivators and uses thereof
WO2020198017A1 (en) * 2019-03-22 2020-10-01 Encoded Therapeutics, Inc. Multiplexing regulatory elements to identify cell-type specific regulatory elements
TWI872078B (zh) * 2019-05-29 2025-02-11 美商編碼製藥公司 用於選擇性基因調節的組合物和方法
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
US12173304B2 (en) 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
EP4591708A2 (en) * 2020-07-08 2025-07-30 Baylor College of Medicine Gene therapy for stxbp1 encephalopathy
CN115867652A (zh) * 2020-07-09 2023-03-28 摩大力斯医疗株式会社 通过靶向mapt基因治疗阿尔茨海默病的方法
US20230279405A1 (en) * 2020-07-24 2023-09-07 University Of Massachusetts Dna-binding domain transactivators and uses thereof
JP2024543001A (ja) * 2021-10-28 2024-11-19 ユーシービー バイオファルマ エスアールエル 核酸構築物、ウイルスベクターおよびウイルス粒子
WO2025212993A1 (en) * 2024-04-05 2025-10-09 Encoded Therapeutics, Inc. Materials and methods for trangene expression in neural cells

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