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

2023-03-07

Abstract excerpt

Merosin-deficient congenital muscular dystrophy (MDC1A) is an autosomal recessive disorder caused by mutations in the LAMA2 gene, resulting in a defective form of the extracellular matrix protein laminin-α2 (LAMA2). Individuals diagnosed with MDC1A exhibit progressive muscle wasting and declining neuromuscular functions. No treatments for this disorder are currently available. We previously showed that postnatal...

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Literature Corpus work
b33c3e4b-c455-55b0-8f1d-759444219faa
DOI
10.1101/2023.03.06.531347
Open publication

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CRISPRa-induced upregulation of human <i>LAMA1</i> compensates for <i>LAMA2</i> -deficiency in Merosin-deficient congenital muscular dystrophyDOI 10.1101/2023.03.06.531347
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