Question

Duchenne's muscular dystrophy is characterized by a lack of dystrophin. When utilizing a KO mouse model, you want to see if introducing the dystrophin

gene can rescue the DMD mouse model. Propose a mechanism to (re)introduce dystrophin back into the KO mouse.

Answer 1

Dystroglycan remains on the sarcolemma in mdx and dystrophin/utrophin double knockout (DKO) mouse muscle and in DMD muscle, although in lower amounts than in normal muscle.

Introducing a new more functional gene into the knockout mouse or repairing the knock out mouse own gene in some manner. Gene therapy using viral vectors and stem cell transplants has been used for exogenous gene delivery.  

Alternatively  introducing proteins that may compensate for dystrophin deficiency in the myofiber (eg, utrophin, biglycan, and laminin), or bolstering the Ko mouse regenerative response.