Muscular dystrophy is the most common fatal genetic condition, especially in children. Doctors in the country have raised hopes for a treatment for muscular dystrophy after mending mutations that cause the disease in dogs. It is said that the landmark study was successful in treating muscle wasting disorder in large mammals. Researchers believe that a clinical trial involving patients with muscular dystrophy would be done soon if the therapy continues to show promise in future animal studies.

1. MS causes
Muscular dystrophy is caused by a mutation that disturbs the normal function of a gene in the X chromosome. Boys are mostly affected by this disease as they have only one X chromosome. As girls have two X chromosomes they have a backup in case one of the genes is damaged. Therefore, girls may not be affected but they could be carriers and may pass on the mutated gene to their children. In muscular dystrophy, the key gene is needed to produce dystrophin, a protein which is important for strong muscle fibers. The protein cannot be made properly if the gene is mutated which causes the muscles throughout the body including the heart, diaphragm, and the skeleton to steadily weaken and waste away. Most of the patients die from breathing or heart problems even before the age of 30.

2. Gene editing
Researchers have used a powerful but experimental gene-editing procedure known as Crispr-Cas9 to correct mutations in the dystrophin gene in a few one-month-old puppies. Harmless viruses were used in this therapy to smuggle the gene-editing molecules into the cells. These viruses once inside, get into the mutated gene and cut it. This causes the cells’ natural repair system to quickly get into action. After Crispr carrying viruses were injected in beagles who carry muscular dystrophy mutation, it resulted in a 60% restoration of normal muscle fibers in 6 weeks.

3. How infusions work
Researchers describe that the infusions had a variable effect on the dog’s muscles. Dystrophin was boosted by just 3% in skeletal muscles. In crucial diaphragm and heart muscles, the levels of dystrophin rose to 58%. It is therefore important to note that intravenous delivery of the virus resulted in significant restoration of dystrophin levels in their heart and diaphragm muscles. This is important in this disease.

4. Noted success
This procedure is said to be extremely promising based on the preliminary findings but to ensure safety and to determine the long term durability of dystrophin expression more work is needed. The results of long term studies in dogs which will assess the efficiency and safety of the procedure would result in determining how soon it can be tested in humans.

5. The future of Crispr-Cas9 technology
The human trial could happen in a few years if everything continues smoothly but will be done with great caution. It is a great breakthrough to see advances in gene-editing applied to Duchenne muscular dystrophy. Larger and longer-term studies would prove if the gene-editing approach would help to slow the progression of the condition and improve muscle strength. This may not be a cure but it is a key step forward in proving that Crispr-Cas9 technology would work to treat people with Duchenne muscular dystrophy.