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Preclinical efficacy and safety of AAVrh10-based plakophilin-2 gene therapy (LX2020) as a treatment for arrhythmogenic cardiomyopathy.

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第一作者： Jing,Zhang

第一单位： Department of Medicine, University of California San Diego, La Jolla, CA, USA.

作者： Jing,Zhang ^[1] ; Erika Joana,Gutierrez-Lara ^[1] ; Aryanne,Do ^[1] ; Lena,Nguyen ^[1] ; Anju,Nair ^[2] ; Nithya,Selvan ^[2] ; Tim,Fenn ^[2] ; Eric,Adler ^[2] ; Richie,Khanna ^[3] ; Farah,Sheikh ^[4]

作者单位： Department of Medicine, University of California San Diego, La Jolla, CA, USA. ^[1] LEXEO Therapeutics Inc, New York, NY, USA. ^[2] LEXEO Therapeutics Inc, New York, NY, USA. rkhanna@lexeotx.com. ^[3] Department of Medicine, University of California San Diego, La Jolla, CA, USA. fasheikh@health.ucsd.edu. ^[4]

DOI 10.1038/s41536-025-00401-6

PMID 40175378

发布时间 2026-03-07

基金项目

P30 NS047101/NS/NINDS NIH HHS/United States R01 HL142251/HL/NHLBI NIH HHS/United States R01 HL162369/HL/NHLBI NIH HHS/United States HL142251/NH/NIH HHS/United States

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NPJ Regenerative medicine

2025年10卷1期

17页

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NPJ Regenerative medicine

2025年10卷1期

17页

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Preclinical efficacy and safety of AAVrh10-based plakophilin-2 gene therapy (LX2020) as a treatment for arrhythmogenic cardiomyopathy.

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Preclinical efficacy and safety of AAVrh10-based plakophilin-2 gene therapy (LX2020) as a treatment for arrhythmogenic cardiomyopathy.

NPJ Regenerative medicine

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NPJ Regenerative medicine

相关期刊

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