CRISPR used to reduce symptoms of Muscular Dystrophy in mice models
CRISPR, or clustered regularly inter-spaced short palindromic repeats, is a technology that allows scientists to make precise alterations to any DNA, whether it be human or bacterial. Most recently, this technology has allowed a team of American, Canadian and Swedish scientists to target the genetic mutation that leads to Muscular Dystrophy. Muscular Dystrophy is an inherited condition that causes progressive weakness and loss of muscle mass, often leading to paralysis. Current treatment for Muscular Dystrophy in humans is essentially limited to mitigating symptoms and trying to maintain mobility and quality of life for the sufferer. As of now, there is no cure for this condition, but this team’s work with mice models may constitute a breakthrough.
Gene editing offers new hope for treating Muscular Dystropy Symptoms
Through CRISPR’s gene editing capabilities, these scientists have found that editing a gene involved in producing proteins that promote muscle strength in their mouse models could reduce their Muscular Dystrophy symptoms. Previously, it had been theorized that reversing the neurodegenerative changes associated with the progression of this disease was impossible, but the team was able to “show that dystrophic features and disease progression were improved and reversed with the treatment was initiated.”
Impact of gene editing
After receiving treatment, the test mice expressed the following:
- Reductions in fibrosis (the formation of excess fibrous connective tissue in an organ or tissue in a reparative or reactive process)
- Larger muscle fiber size (which reduced or in some cases prevented symptoms from occurring)
- In mice that had already begun to experience paralysis, the treatment allowed them to stand up and move around by their own volition
- Nerve conduction speed increased (a sign that they myelin coating destruction was being reversed)
Collectively, these findings may enable mutation-independent treatment for all sufferers of Muscular Dystrophy. And, treating Muscular Dystrophy is only the beginning—this therapeutic strategy may be applicable to many inherited and acquired diseases. Only the future knows what CRISPR technology means for the future of previously untreatable diseases.
If you are a WorldCare member and have recently been diagnosed with Muscular Dystrophy and would like a medical second opinion to ensure your diagnosis is accurate and that you are on the optimal treatment pathway, please request service today.