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Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model.
Mol Genet Metab. 2023 Jan;138(1):106982. doi: 10.1016/j.ymgme.2022.106982. Epub 2022 Dec 23.
Mol Genet Metab. 2023.
PMID: 36580829
Free PMC article.
Characterization of variants of uncertain significance in isovaleryl-CoA dehydrogenase identified through newborn screening: An approach for faster analysis.
D'Annibale OM, Koppes EA, Alodaib AN, Kochersperger C, Karunanidhi A, Mohsen AW, Vockley J.
D'Annibale OM, et al. Among authors: kochersperger c.
Mol Genet Metab. 2021 Sep-Oct;134(1-2):29-36. doi: 10.1016/j.ymgme.2021.08.012. Epub 2021 Aug 30.
Mol Genet Metab. 2021.
PMID: 34535384
Free PMC article.
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An acyl-CoA dehydrogenase microplate activity assay using recombinant porcine electron transfer flavoprotein.
Zhang Y, Mohsen AW, Kochersperger C, Solo K, Schmidt AV, Vockley J, Goetzman ES.
Zhang Y, et al. Among authors: kochersperger c.
Anal Biochem. 2019 Sep 15;581:113332. doi: 10.1016/j.ab.2019.06.003. Epub 2019 Jun 10.
Anal Biochem. 2019.
PMID: 31194945
Free PMC article.
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Mitochondrial energetics is impaired in very long-chain acyl-CoA dehydrogenase deficiency and can be rescued by treatment with mitochondria-targeted electron scavengers.
Seminotti B, Leipnitz G, Karunanidhi A, Kochersperger C, Roginskaya VY, Basu S, Wang Y, Wipf P, Van Houten B, Mohsen AW, Vockley J.
Seminotti B, et al. Among authors: kochersperger c.
Hum Mol Genet. 2019 Mar 15;28(6):928-941. doi: 10.1093/hmg/ddy403.
Hum Mol Genet. 2019.
PMID: 30445591
Free PMC article.
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