34 research outputs found
CRISPR/Cas9 gene-editing platform corrects mutations associated with Duchenne muscular dystrophy
No full textAdenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
No full textMethods for Scarless, Selection-Free Generation of Human Cells and Allele-Specific Functional Analysis of Disease-Associated SNPs and Variants of Uncertain Significance
Get PDFA correction to this article has been published and is linked from the HTML and PDF versions of this paper. The error has not been fixed in the paper
Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy
No full text
Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells
No full text
