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Gene Therapy Helps Weak Mice Grow Strong
Transcript
Conclusion
Was the study presented clearly?
- yes, but I did have to use an other source to help support the information the author was giving because it was vague.
The author's conclusion and future experiments:
- It concludes that the therapy isn't "one size fits all", but this therapy does show promise for at least some of the neuromuscular disorders.
- Researches plan on continuing their work by looking into other neuromuscular diseases in mice to see which ones this gene therapy will help.
Why did I choose this article?
- Millions of people are affected by neuromuscular disorders and about 40% of them are under the age of 18
- No one should have to live with this.
Results
- After just seven weeks the mice that had other neuromuscular disorders gained weight and their muscles grew stronger.
- The mice with defective DOK7 genes were severely underweight and died at a young age.
- For the mice with EDMD the size of the NMJs increased compared to the mice with EDMD that were not treated.
- Unfortunately, the mice with EDMD did not fully recover like the mice with other disorders did.
The Study
- A team of researchers from the University of Tokyo, led by molecular biologist Yuji Yamanashi, engineered a virus to contain a effective normal human DOK7 gene.
- Researches injected the virus into mice with the two neuromuscular diseases, which shuttled the gene into the cells.
- Researchers began to try their experimental virus on mice with EDMD.
Background Information
- Muscular Dystrophy- a disease that creates muscle loss and over time becomes a lot worse.
- Amyotrophic Lateral Sclerosis (ALS)- a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement.
- Neuromuscular Junction (NMJ)- interface between neurons and muscle fibers.
- DOK7- is a protein used for the proper formation of NMJ.
MAIN QUESTION:
- Will a virus coded with effective DOK7 protein help improve muscles in subjects with certain neuromuscular disorders?
- Will DOK7 gene therapy help patients with different muscular diseases such as Emery-Dreifuss Muscular Dystrophy (EDMD)?
Gene Therapy Helps Weak Mice Grow Strong
By: Jocelyn Kaiser
Source: news.sciencemag.org
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