Gene Editing Can Help Treat Duchenne Muscular Dystrophy

A new research at the Duke University has successfully treated an adult mouse model suffering from Duchenne Muscular Dystrophy (DMD) using CRISPR-a gene editing technique. It is the first time that the technique has been successful in treating a genetic disease of a developed living mammal. The researchers believe that these new developments indicate that the technique has the potential to work with equal efficacy on human beings.

The experiments using CRISPR have been going on for more than decades, but the progress has been snail-paced. However, with the newfound success, researchers are now ready to take a substantial step ahead and develop a possible treatment for treating DMD in fully-grown humans. Earlier the Duke University researchers had used the CRISPR technique for correcting the genetic mutations of Duchenne patients in their cultured cells whereas other labs also had tried correcting the single-cell embryo genes within the laboratory environment.

The single-cell method is currently considered unethical for working on humans whereas the genetic mutation technique is hard to implement, as it requires a transfer of the treated cells back to the tissues in the muscles.

DMD-progressive fatal disease

DMD is an incurable as well as highly devastating progressive disease in which the muscles degenerate resulting in disability in boys at a tender age of 10 years or less followed by death due to breathing difficulties or heart failure. Causes of DMD include the presence of a defective gene in the body due to which it cannot encode, dystrophin, a type of protein necessary for proper functioning of the muscle.

Researchers have been trying for long to find a solution for treating this common hereditary illness through gene therapy, however, until now had little success. Although, as an alternative option, antisense oligonucleotides, a chemical drug is undergoing clinical trials, the latest success of CRISPR with the gene technique in treating DMD in the adult mouse has made the scientists more hopeful about its ability to treat the disease in humans as well.