ETH Zurich molecular biologist Mandy Boontanrart is researching gene therapies that could be used to cure two of the most common types of inherited anaemia. She has now developed a promising approach for so-called beta-hemoglobinopathies.
Many hereditary diseases have largely been considered to be incurable. The intervention in the genome is too unpredictable and complex and the results of this manipulation too uncertain. This is because these diseases often involve not just one but several genes, which can be located on different chromosomes.
But with the phenomenal uptake of the CRISPR-Cas9 gene scissors, the rulebook has been extensively rewritten. In just the past few years, targeted manipulation of individual genes or even entire building blocks of DNA has come a very long way. The results of these collaborative efforts is that curing hereditary diseases in humans is now within reach.