Hereditary amyloidosis is a rare genetic disease. This disease is characterised by the unusual deposition of protein clusters in tissues, between cells, and often in the walls of small vessels. The first symptoms appear in those aged between 20 and 40 and are fatal within 10 years. If you have the disease, there is a 50/50 chance that you will pass it on to your children.
Professor Ed Gane
Ed Gane is an exceptional New Zealand professor who helped create the national liver transplant unit. He is also the pioneer in curing hepatitis C through his worldwide drug trials.
The professor has now taken on a new challenge: trying to cure amyloidosis, a disease that affects around 50 New Zealand families. These families are now being accompanied by Gane throughout their healing process: from diagnosis to liver and heart transplants, the professor helps them, talks to them, holds their hands and is more than there for them.
Clinical trials by professor
Two years ago, Gane was able to try and achieve his goal when he was offered the opportunity to carry out the first human trial of a new medical technology enabling scientists to modify defective DNA genes.
CRISPR gene editing (short palindromic repeats regularly spaced in a cluster) is a natural process that has evolved to protect bacteria from viruses. A major medical breakthrough!
Professor Gane had the honour of being able to use this new advance on 12 of his amyloidosis patients. Nobody knew whether this trial would be a success, but two years on, the initial results indicate that all the participants will be cured of this fatal disease, which represents real hope for the children of people carrying the amyloidosis gene.
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