Sentences with phrase «delivering dystrophin»

They usually die of heart failure or lung disease, so delivering dystrophin to the skeletal muscles — even if that were possible — would not help much.

Not exact matches

Gersbach and his team first delivered the therapy directly to a leg muscle in an adult mouse, resulting in the restoration of functional dystrophin and an increase in muscle strength.

The therapy delivered directly to a leg muscle restored dystrophin and increased muscle strength in the adult mouse, after which the researchers injected a combination of CRISPR and AAV, the most popular virus today for delivering genes, into the bloodstream to reach every muscle.

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.

The webinar will cover different virus types, understanding the screening process, dosing, dystrophin constructs, promoters, and how does the virus and gene that is delivered integrate into the muscle cell.