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Therapeutic Genetics and Disease Modeling in LAMA2-CMD

CRISPRa-induced upregulation of human LAMA1 compensates for LAMA2-deficiency in Merosin-deficient congenital muscular dystrophy

Annie I. Arockiaraj, Marie A. Johnson, Anushe Munir, et al. (2023). bioRxiv. Cited 3 times. https://doi.org/10.1101/2023.03.06.531347