Muscular dystrophy is a heterogeneous muscular degenerative disease characterised by weakening of the skeletal muscle, poor balance and in extreme, but common circumstances, the loss of a person’s ability to move. At present there is no known cure for this genetic disorder however recent research has given sufferers hope. Studies conducted by Odom et al, have shown that viral mediated gene therapy can halt the progression of muscular dystrophy, in particular the use of adeno-associated viruses have delivered encouraging results. The attractive feature of using adeno-associated viruses(AAV) is the small size of the virus. AAVs immediate very low immunogenicity, seemingly confined to the generation of neutralizing antibodies, while they cause no clearly-defined cytotoxic response. This feature, along with the ability to infect both dividing and non-dividing cells, presents their dominance as use in vectors for muscular dystrophy treatment.
There are however disadvantages to using AAV’s. Tests carried out by Odom et al, suggest some therapeutic genes that are used to treat muscular dystrophy, require the complete transcription onto the virus’s 4.8 kilobase genome. Currently due to their limited vector capacity, this cannot be done. The scientists are therefore researching ways of expanding the limited coding capacity of the AAV’s. One option suggested by the study is the idea of forming the inverted terminal repeats of the AAV’s to form head to tail concatamers, which would result in almost doubling the capacity of the vector. As advocated by Odom et al, more research is essential in order to overcome the disadvantages of using AAV’s in muscular dystrophy treatment, before they can be used clinically as a treatment.
Source of Article : http://www.pubmedcentral.nih.gov/picrender.fcgi?artid=1894910&blobtype=pdf&tool=pmcentrez
