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AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia.
Chang JC, Ryan MR, Stark MC, Liu S, Purushothaman P, Bolan F, Johnson CA, Champe M, Meng H, Lawlor MW, Halawani S, Ngaba LV, Lynch DR, Davis C, Gonzalo-Gil E, Lutz C, Urbinati F, Medicherla B, Fonck C. Chang JC, et al. Among authors: ngaba lv. Mol Ther Methods Clin Dev. 2024 Jan 22;32(1):101193. doi: 10.1016/j.omtm.2024.101193. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38352270 Free PMC article.
Frataxin controls ketone body metabolism through regulation of OXCT1.
Dong YN, Mesaros C, Xu P, Mercado-Ayón E, Halawani S, Ngaba LV, Warren N, Sleiman P, Rodden LN, Schadt KA, Blair IA, Lynch DR. Dong YN, et al. Among authors: ngaba lv. PNAS Nexus. 2022 Jul 26;1(3):pgac142. doi: 10.1093/pnasnexus/pgac142. eCollection 2022 Jul. PNAS Nexus. 2022. PMID: 36016708 Free PMC article.
Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia.
Mercado-Ayón E, Warren N, Halawani S, Rodden LN, Ngaba L, Dong YN, Chang JC, Fonck C, Mavilio F, Lynch DR, Lin H. Mercado-Ayón E, et al. Among authors: ngaba l. Front Neurosci. 2022 Mar 24;16:819569. doi: 10.3389/fnins.2022.819569. eCollection 2022. Front Neurosci. 2022. PMID: 35401081 Free PMC article.