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5.10 Myasthenia gravis In contrast to the conventional DNA-based approach, autologous BCMA RNA chimeric antigen receptor T cells (BCMA rCAR-T), as exemplified in the Descartes-08 trial, resulted in clinically meaningful reductions in all measures of myasthenia gravis severity, including the induction of minimal symptom expression and the elimination of dependence on intravenous immunoglobulin infusions in adults with generalized myasthenia gravis (NCT04146051) (135)

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These mechanisms represent potential therapeutic targets for CF treatment through modulation of post-translational modifications in the CFTR maturation pathway (Zaman et al., 2019)