LX2020

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LX2020 is an experimental gene therapy for arrhythmogenic cardiomyopathy caused by mutations to the plakophilin-2 gene. It is delivered via adeno-associated virus and was developed by Lexeo Therapeutics.[1][2][3]

References[edit]

  1. ^ Sheikh, Farah; Zhang, Jing; Gutierrez Lara, Erika; Do, Aryanne; Nguyen, Lena; Bradford, William H.; Nair, Anju; Selvan, Nithya; Gutierrez, Sonia; Law, Ken; Fenn, Tim; Khanna, Richie (2023). "Abstract 18583: Preclinical Efficacy and Safety of Intravenously Administered LX2020 Gene Therapy for the Treatment of Arrhythmogenic Cardiomyopathy". Circulation. 148. doi:10.1161/circ.148.suppl_1.18583. S2CID 266416146.
  2. ^ Sheikh, Farah; Zhang, Jing; Wang, Jie; Bradford, William H; Nair, Anju; Fargnoli, Anthony; Selvan, Nithya; Gutierrez, Sonia; Law, Ken; Fenn, Tim; McCormac, Paul; Barth, Jay A; Khanna, Richie (8 November 2022). "Abstract 13599: LX2020, an Adeno Associated Viral-Based Plakophilin 2 Gene Therapy Stabilizes Cardiac Disease Phenotype in a Severe Mouse Model of Arrhythmogenic Right Ventricular Cardiomyopathy". Circulation. 146 (Suppl_1). doi:10.1161/circ.146.suppl_1.13599. ISSN 0009-7322. S2CID 259475762.
  3. ^ Argirò, Alessia; Ding, Jeffrey; Adler, Eric (December 2023). "Gene therapy for heart failure and cardiomyopathies". Revista Española de Cardiología (English Edition). 76 (12): 1042–1054. doi:10.1016/j.rec.2023.06.009. PMID 37506969.