Isciii Advances Towards a Possible Gene Therapy with the Crispr Tool for a Rare Unhealed Muscle Disease

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The study, led by a team from the Institute for Rare Disease Research (IIER), has been published in the journal Molecular Therapy Advances. The results of this work, carried out in cell and animal models, suggest an increase in survival in this uncured pathology, and represent the first therapeutic validation of a gene editing strategy mediated by CRISPR-Cas9 to treat L-CMD. Congenital muscular dystrophy associated with the LMNA gene is a rare g…

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Research by the Carlos III Institute of Health (ISCIII) has used the CRISPR genetic editing tool to explore a possible treatment for a very rare and uncured muscle disease, congenital muscular dystrophy associated with the LMNA gene (abbreviated as L-CMD).