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

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 ne...

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Detalles Bibliográficos
Autores principales: Arockiaraj, Annie I., Johnson, Marie A., Munir, Anushe, Ekambaram, Prasanna, Lucas, Peter C., McAllister-Lucas, Linda M., Kemaladewi, Dwi U.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10028808/
https://www.ncbi.nlm.nih.gov/pubmed/36945402
http://dx.doi.org/10.1101/2023.03.06.531347

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