Sentences with phrase «editing of the dystrophin»
First, CRISPR would need to be delivered to both cardiac and skeletal muscle cells, where precision editing of the dystrophin gene would take place, with minimal risk of off - target editing.
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«CRISPR - Cpf1 gene - editing can be applied to a vast number of mutations in the dystrophin gene.
«This work demonstrates the feasibility of using a single gene editing platform, plus the regenerative power of stem cells to correct genetic mutations and restore dystrophin production for 60 percent of Duchenne patients,» said Pyle, associate professor of microbiology, immunology and molecular genetics and member of the Broad Stem Cell Research Center.
These splice sites instruct the genetic machinery to build abnormal dystrophin molecules, but once the gene is successfully edited it expresses a much - improved dystrophin protein product, enhancing the function of the muscle tissue.
Scientists say the new strategy enhances the accuracy for surgical - like editing of the human genome, correcting mistakes in the DNA sequence that cause devastating diseases like DMD, a deadly condition caused by defects in the dystrophin gene.
Multiplex CRISPR / Cas9 - based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.
Although Nelson et al. observed only 2 % genome editing in one experiment, they found the exon - skipped transcript constituted 59 % of total dystrophin mRNA, similar to the 39 % observed by Tabebordbar et al..