Technological Breakthrough for DMD Research

According to News Medical, a team of researchers from London have created new technologies that may aid in further understanding Duchenne muscular dystrophy (DMD). Not only this, but the new development could also assist in testing for possible drugs for DMD as well.

Duchenne muscular dystrophy (DMD) is one out of nine forms of muscular dystrophy. In those with DMD, the body is unable to make dystrophin in the muscles. As a result, the muscles are weakened and this eventually leads to heart and respiratory weakness as well. Typically, DMD symptoms begin in early childhood. Among these symptoms are muscle weakness beginning in the legs, difficulty with motor skills, frequent falling, fatigue, learning disabilities and low IQ, and more. DMD is inherited and there are a handful of treatments to manage symptoms of the disease. That said, there is no cure for DMD yet. To read more about the condition, click here.

Though there is no effective cure for DMD at this time, this new research is definitely an advancement to take note of.

The research team is planning on using a certain type of stem cell that is re-programmed into an embryonic state. By utilizing this, these cells are able to grow and become any cell in the human body. In this specific scenario, researchers are growing these into skeletal and cardiac muscle cells.

The challenge in this type of research, though, is that the muscle cells of each donor have different genetic makeups. To fix this inconsistency, the team uses a gene editing tool called CRISPR. This tool is a well-known and widely used technology in research that can manipulate specific sections of DNA with great accuracy.

In this instance, researchers are utilizing CRISPR to edit the genetic code in DMD patients. Specifically, CRISPR is being used in their stem cells to “correct” the genetic code and put in place a healthy muscle cell. This muscle cell should still have the same genetic make-up of the donor patient’s cell.

This is a great feat for research in this area, and it is a huge step forward in understanding more about DMD and potentially pinpointing a new treatment for the disease.

“Dystrophin is the largest gene in humans, comprising 2.4 million base-pairs and 79 separate protein-coding regions. This makes it one of the most difficult genes to correct, but we’ve managed to do it,” said Dr. Yung-Yao Lin, who helped lead the research project.

“We can now use DMD patient skin cells which are stored in the biobank, and turn them into stem cells to generate an unlimited supply of skeletal and cardiac muscle cells in culture dishes. Our genetically-corrected stem cells will also help us establish a cell-based platform to test future drug candidates.”

This breakthrough in medical technology for research in DMD is great news for the DMD community as well as the scientific community. Utilizing CRISPR like never before, scientists may be able to develop brand new therapeutic options for those that suffer from DMD.

 


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