Potential Disease Targets for Gene Therapy

Another, relatively common, genetic disease that has the potential to be treated hy gene therapy is Duchenne muscular dystrophy (DMD). Most effort to replace the defective DMD gene has concerned an ex vivo strategy to transfect isolated muscle cells with multiple copies of the DMD gene and re-implant the cells back into the patient s muscle. There has so far been little work on the in vivo delivery of the DMD gene as there are few vectors suitable for the efficient delivery of genes into muscle cells in vivo and ensure tissue specific expression . 

Cancer is the second commonest cause of death in the industrialised world. In the UK over a third of people will develop cancer at some point in their lifetime, and around half of cancers are not curable using the traditional treatments of surgery, radiotherapy or chemotherapy. Gene therapy has emerged as a potential alternative to the existing treatments, and its potential is such that protocols for the treatment of cancer now account for over 80% of the gene therapy clinical trials.  

There are numerous approaches to using gene therapy to treat cancer, and some of these are outlined below. 

Other diseases that have been investigated for possible treatment with gene therapy include AIDS , neurodegenerative diseases such as Alzheimer s and Parkinson s, traumatic nerve injury and autoimmune diseases .  

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