Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
... important proof of concept for future preclinical and clinical work.
Find Plasmids for Disease Research...research, researchers are eager to apply it in a clinical setting. CRISPR is often used for animal germline...exon skipping is a good option for DMD treatment. Clinical trials have used oligonucleotide exon skipping...rate of exon 51, which resulted in appropriately localized dystrophin. In a subsequent study, they used CRISPR...six months.
Future directions and obstacles to clinical CRISPR editing
Given the success of the CRISPR-mediated...be evaluated separately, increasing the time to clinical approval.
Even with the challenges precision editing...become one of the first diseases to be treated clinically with CRISPR. Many obstacles remain for CRISPR...