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ELP1, the Gene Mutated in Familial Dysautonomia, Is Required for Normal Enteric Nervous System Development and Maintenance and for Gut Epithelium Homeostasis.
Chaverra M, Cheney AM, Scheel A, Miller A, George L, Schultz A, Henningsen K, Kominsky D, Walk H, Kennedy WR, Kaufmann H, Walk S, Copié V, Lefcort F. Chaverra M, et al. Among authors: lefcort f. J Neurosci. 2024 Sep 11;44(37):e2253232024. doi: 10.1523/JNEUROSCI.2253-23.2024. J Neurosci. 2024. PMID: 39138000
Gut microbiome dysbiosis drives metabolic dysfunction in Familial dysautonomia.
Cheney AM, Costello SM, Pinkham NV, Waldum A, Broadaway SC, Cotrina-Vidal M, Mergy M, Tripet B, Kominsky DJ, Grifka-Walk HM, Kaufmann H, Norcliffe-Kaufmann L, Peach JT, Bothner B, Lefcort F, Copié V, Walk ST. Cheney AM, et al. Among authors: lefcort f. Nat Commun. 2023 Jan 13;14(1):218. doi: 10.1038/s41467-023-35787-8. Nat Commun. 2023. PMID: 36639365 Free PMC article.
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F. Schultz A, et al. Among authors: lefcort f. bioRxiv [Preprint]. 2023 May 24:2023.05.22.541535. doi: 10.1101/2023.05.22.541535. bioRxiv. 2023. Update in: Sci Rep. 2023 Oct 30;13(1):18600. doi: 10.1038/s41598-023-45376-w PMID: 37293016 Free PMC article. Updated. Preprint.
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F. Schultz A, et al. Among authors: lefcort f. Sci Rep. 2023 Oct 30;13(1):18600. doi: 10.1038/s41598-023-45376-w. Sci Rep. 2023. PMID: 37903840 Free PMC article.
Metabolic Deficits in the Retina of a Familial Dysautonomia Mouse Model.
Costello SM, Schultz A, Smith D, Horan D, Chaverra M, Tripet B, George L, Bothner B, Lefcort F, Copié V. Costello SM, et al. Among authors: lefcort f. Metabolites. 2024 Jul 31;14(8):423. doi: 10.3390/metabo14080423. Metabolites. 2024. PMID: 39195519 Free PMC article.
57 results