Instead, rare diseases found in children can be treated by treating the gene that is causing the disease.
“Inside every cell we have a nucleus, and inside it there are 23 pairs of chromosomes, and inside it there are 25,000 genes that control everything that we are: from how tall we are to the colour of our hair,” he said.
“Just one single gene mistake can cause a devastating disease. And if we can understand what that gene is we can understand [how to treat it].”
Professor Gaspar cited an inspiring examples of a gene therapy success story, while showing a picture of a young child from Poland.
“This is a little girl back in May 2013. A single gene in her bone marrow meant that she couldn’t produce any white cells, which are there to protect us from bacteria and viruses.
As the girl lacked any white cells, even a cold could be life threatening.