In 1986, Louis Kunkel is recognized for discovering the dystrophin gene...


He was not the only individual focused on finding the gene responsible for Duchenne. There were several groups of investigators in Europe, Canada, and the US racing to find this, the first gene to be identified. Dystrophin - thought to be the holy grail, the key that would soon unlock treatments and a cure for Duchenne.


While attending a meeting in Australia, one young researcher stood up and said "We have the gene, we know the protein product. We have a virus (the AV) and we will just deliver it!" We all locked hands and smiled, sure that this would indeed be the case.


Too new to the world of advocacy at that time, none of us asked "How? While the AV has the capacity to hold the very large protein product, is it toxic? How will your ensure delivery to every muscle in the body?"


The devil is always in the details and now 25 years later, we are making slow, deliberate steps forward, into single muscles and next, to a single limb. This is not the progress we had expected or hoped to achieve, but it is progress nonetheless.


The articles below are interesting insights into gene therapy, and will give you a good sense of the history of this therapy:


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