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Bio 198 Gene Therapy Paper Final Edition Bruce Zhang by no.deuterostome is a document available to read on EtoBox.
Duchenne Muscular Dystrophy (DMD) is a severe, progressive muscle-wasting disease caused by mutations in the dystrophin gene, primarily affecting males. Recent advancements in gene therapy, including Eteplirsen and CRISPR/Cas9 technology, aim to induce dystrophin production or modify the gene, but challenges remain in achieving permanent cures and addressing ethical concerns. The innovative CRISPR-associated transposase (CAST) system shows promise for directly inserting the full-length dystrophin gene into
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- no.deuterostome
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- EN