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Our findings may indicate reduced CPT-I activity in the patient group, which is essential for the -oxidation of long-chain fatty acids, facilitating their conversion into acylcarnitines for transport from the cytosol into the inner mitochondrial matrix [17,18]

doi: 10.1002/imt2.43 24 CarlsonDBMcFaddenJWDAngeloAWoodworthJCDrackleyJK

First, we used CMS4-derived organoids (from a patient with PM) in which the catalytic subunit of glutamate-cysteine ligase (GCLC) was deleted using CRISPR-CAS9 technology

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