Genetic Editing to Fight Muscular Dystrophy

New ground is being broken by CRISPR on the frontier of genetic editing. A recent study out of Texas may prove to be one of the first steps in implementing this new technology in the manipulation of human genes to fight life-threatening diseases

One Protein Can Cause a Collapse

For those diagnosed with Duchenne Muscular Dystrophy, life can be bleak. The condition is a progressive one, and those who suffer from it begin to slowly lose control of their body as they age and their muscles deteriorate around them. Linked to a missing gene known as exon 44 the disease cripples the body by way of interfering with a protein known as dystrophin in muscle cells. Without the integrity of this protein, muscles struggle to maintain their shape and elasticity leading to loss and eventual withering. It’s a lifetime disability and has no known cure, until perhaps now. 

genetic editing

Genetic Editing Could Fill Missing Piece in Fight Against Muscular Dystrophy 

The proof of concept was demonstrated by extracting heart tissues from those suffering from DMD and correcting a small sample size of cells. From then on it moved to live biology and the research team, through painstaking effort and clever science, demonstrated its success in mice. In simple terms, it all boils down to the one gene exon 44. In patients who suffer from the disease the gene is simply missing, but with CRISPR the absent gene can be inserted back into the DNA of a stem cell in which the muscles of the body will be built from. The rebuild has shown initial success and has received a grant from Parent Project Muscular Dystrophy to continue pursuing research.

genetic editing

The method works to a larger benefit than treatments that exist today which involve signaling to the body to skip over the instructions of the missing gene but naturally, with limited instructions, results are mixed.

Genetic disorders are going to be a hotbed of development in the CRISPR field as faulty DNA lies at the heart of these diseases. With the ability to modify and manipulate this study will undoubtedly be the first of many to come with the potential to not only save lives but also vastly improve the quality of those affected by debilitating genetic diseases like DMD.  



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