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

Annie I Arockiaraj,Marie A Johnson,Anushe Munir,Prasanna Ekambaram,Peter C Lucas,Linda M McAllister-Lucas,Dwi U Kemaladewi

bioRxiv : the preprint server for biology（2023）

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摘要

Merosin-deficient congenital muscular dystrophy (MDC1A) is an autosomal recessive disorder caused by mutations in the LAMA2Lama1Lama2LAMA1LAMA1LAMA2LAMA2SaLAMA1LAMA1FGFR2, TGF-2, and ACTA2LAMA1

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