Giulia Maule, doctoral student in Biomolecular sciences at the University of Trento, was awarded – with her research group – the Klosterfrau Award 2020 for the study that appeared in Nature Communication "Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing".
The Klosterfrau foundation supports young scientists worldwide in their research work to understand the causes of diseases and to find new therapies to treat pediatric respiratory diseases. These diseases are among the most common in childhood and take a heavy toll on both the patients and their families.
The research project is the result of collaboration between a team of researchers of Cibio department of UniTrento, led by Anna Cereseto, and KU Leuven in Belgium, in the framework of a 2-year study funded by Fondazione Ricerca Fibrosi Cistica (project FFC#1/2017 SpliceFix: fixing splicing defects in the CFTR gene through CRISPR/Cas9 technology).
This new approach opens new perspective in the treatment of cystic fibrosis, a genetic disorder for which there is currently no cure. Using the CRISPR-Cas technology, known as molecular scissors, researchers were able to edit two of the mutations responsible for cystic fibrosis.