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Genetic Engineering Publications - GEG Tech top picks
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BigField GEG Tech's insight:
The study was the first to create corrected human iPS cells that could directly restore functional muscle tissue affected by the disease. Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy. The approach uses the CRISPR/Cas9 technology to correct genetic mutations that cause the disease. The study, which was led by co-senior authors April Pyle and Melissa Spencer and first author Courtney Young, was published in the journal Cell Stem Cell. 
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BigField GEG Tech's insight:
In this study, the authors established a protocol for differentiating mouse and human pluripotent stem cells into muscle. Their strategy provides an attractive model to study the origin of the pathological defects associated with DMD.
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Clinical Implications of Basic Research from The New England Journal of Medicine — The CRISPR Way to Think about Duchenne’s
BigField GEG Tech's insight:
Duchenne's muscular dystrophy is caused by a loss-of-function mutation in DMD. Studies of the CRISPR–Cas9 method of excising the mutated region show efficacy in a mouse model of the disease.
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BigField GEG Tech's insight:
In this study, adeno-associated virus was used to deliver the CRISPR/Cas9 system to the mdx mouse model of DMD to remove the mutated exon 23 from the dystrophin gene. This includes local and systemic delivery to adult mice and systemic delivery to neonatal mice. Exon 23 deletion by CRISPR/Cas9 resulted in expression of the modified dystrophin gene, partial recovery of functional dystrophin protein in skeletal myofibers and cardiac muscle, improvement of muscle biochemistry, and significant enhancement of muscle force. This work establishes CRISPR/Cas9-based genome editing as a potential therapy to treat DMD.
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An HDR-independent therapeutic genome-editing approach corrected the splice-site mutation in Lama2 in a mouse model of congenital muscular dystrophy type 1A, and may be applied more broadly to correct splice-site mutations associated with other diseases.