The Promising Future of Gene Therapy with Duchenne Muscular Dystrophy

Involving animals as test subjects is not new to medical research since the functioning of their cells and organs closely resembles the functioning of human's cells and organs. This is why some mice, cats, frogs, pigs, primates, and even dogs are sometimes bred specifically for medical research. Animals can also be vital in bringing incredible medical progress to understanding and treating human conditions like, for example, Duchenne muscular dystrophy.

Muscular dystrophy literally translates to ‘badly nourished muscles’ with dystrophy coming from the roots ‘dys’ (bad) and ‘trophy’ (nourished). Among the nine types of muscular dystrophy is Duchenne Muscular Dystrophy (DMD). DMD is an incurable muscle-wasting disorder resulting from mutations in the dystrophin gene. Basically, the muscles lack a protein responsible for muscle strengthening (dystrophin), and the body cannot keep up with the necessary cell repair. Progressively, this results in muscle degeneration and weakness.

Research findings show that the disease commonly affects young boys and is rarer among girls. Regardless of gender, it seriously affects the lives of the patients and the people around them. At a young age, DMD patients have trouble in performing even simple tasks like walking, climbing, running, or getting up. When in school, they may find it hard to keep up with their classmates upon doing physical tasks, not to mention tasks that involve cognition and behavior as some patients were found to have a learning disability. Heartbreakingly, the disease will soon result in more serious and life-threatening complications like scoliosis, respiratory problems, and heart disease.

Living a life similar to those of DMD patients is simply incomprehensible. Support from the family, physical therapy exercises, and medications are some known ways to help ease the suffering. Fortunately, scientists and medical experts are continually conducting medical research and clinical trials to ameliorate the patient's’ conditions.

Duchenne MD Research and Findings

Intensive research dedicated to understanding the causes and unraveling effective treatments to DMD has untiringly been conducted by scientists around the globe. Millions of dollars have been donated and invested in these endeavors in the hopes of finally putting an end to this debilitating condition. Scientists are particularly looking into developing treatments that could protect muscles from injury, regrow or repair muscles and replace the faulty dystrophin genes. One of the newest research development is on gene transfer as a therapeutic agent. 

This current study was conducted by Royal Holloway researchers in London. The goal was to introduce a microdystrophin gene therapy to 12 Golden Retriever Labradors, who also suffered from DMD before testing it on humans. Normally, humans suffering from DMD do not live past 20 years. As for the dogs, they were expected to live for a shorter time period of 6 months.