Gene therapy against neuromuscular diseases

Stephen Hawking, who is diagnosed with ALS (amyothophic lateral sclerosis)

So, no need for more Ice Bucket Challanges ? maybe not quite, but we're getting there! 
A virus that shuttles a therapeutic gene into cells has strengthened the muscles, improved the motor skills, and lengthened the lifespan of mice afflicted with two neuromuscular diseases. The approach could one day help people with a range of similar disorders, from muscular dystrophy to amyotrophic lateral sclerosis, or ALS

Many of these diseases involve defective neuromuscular junctions—the interface between neurons and muscle cells where brain signals tell muscles to contract. In one such disease, a form of familial limb-girdle myasthenia, people carry two defective copies of the gene called DOK7, which codes for a protein that’s needed to form such junctions

In the new study, researchers led by molecular biologist Yuji Yamanashi of the University of Tokyo first injected young mice engineered to have defective Dok7 with a harmless virus carrying a good copy of the Dok7 gene, which is expressed only in muscle. Within about 7 weeks, the rodents recovered.

#ReadMore of this story in: http://news.sciencemag.org/biology/2014/09/gene-therapy-helps-weak-mice-grow-strong