WebSep 21, 2024 · Previously, we and others used CRISPR/Cas9-mediated genome editing to permanently correct dystrophin mutations in mouse models of DMD and patient-derived … WebJun 16, 2024 · By CRISP/Cas9-based genome editing, we corrected the dystrophin mutation in expanded MuSCs and restored the skeletal muscle dystrophin expression upon transplantation in mdx mice. Our studies established a reliable and feasible platform for gene correction in MuSCs by genome editing, thus greatly advancing tissue stem cell …

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WebDifferent timing and injection methods restored dystrophin protein expression in cardiac and skeletal muscle to varying degrees from 3 to 12 weeks after injection. Together, these studies support further research into the potential for CRISPR/Cas9 genome editing to treat DMD and possibly other genetic diseases. WebJan 22, 2016 · Each of these methods restored dystrophin protein expression in cardiac and skeletal muscle to varying degrees, and expression increased from 3 to 12 weeks after injection. Postnatal gene editing also enhanced skeletal muscle function, as measured by grip strength tests 4 weeks after injection. fly fishing switzerland

Long-term evaluation of AAV-CRISPR genome editing for Duchenne …

WebApr 12, 2024 · Background Mutations in the DMD gene encoding dystrophin—a critical structural element in muscle cells—cause Duchenne muscular dystrophy (DMD), which … WebIn particular, CRISPR-Cas9 gene editing components packaged by self-complementary AAV (scAAV) demonstrate robust viral transduction and efficient gene editing, enabling restoration of dystrophin expression throughout skeletal and cardiac muscle in animal models of DMD. Using this AAV9-intein-split Cas9 approach with two gRNAs at 2 × 1013 vgs/kg, intramuscular injection revealed a robust, local response with dystrophin protein levels up to 32% of wildtype, which sufficed to improve muscle fiber features such as ferret diameter and proportion of centralized nuclei [48]. A high … See more The out-of-frame mutation inflicted by the absence of exon 52 in our pig model suited well for therapy by an additional Cas9-induced snipping of exon 51, for which two gRNAs … See more Although Duchenne’s muscular dystrophy is a disabling and immobilizing disease with a shortened life span and grave implications with … See more In the light of emerging new therapies, health economic questions might arise; our group investigated the cost of illness (COI) of DMD and the milder allelic BMD from a socio-economic and clinical perspective in … See more fly fishing syracuse ny