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Gene editing in cells has revolutionized the paradigm of modern cell biology. The ease of use of the CRISPR/Cas9 system for gene manipulation has led to rapid and wide adoption across many fields, including biological research, biotechnology and medicine. This technology allows gene modification by producing a specific double stranded cut in the DNA and the repair by homology-independent targeted insertion (HITI) of DNA. We used CRISPR/CAS9 to modify specific mutations in-vitro in a muscular disorder to show its potential use as a therapeutic tool.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research