Martino L. Morella

Apr 12, 2023 | nature.com | Vinod Udayar |Martino L. Morella |Verdon Taylor |David J. Vocadlo |Alexandra Gehrlein |Martin Ebeling

AbstractMutations in glucocerebrosidase cause the lysosomal storage disorder Gaucher’s disease and are the most common risk factor for Parkinson’s disease. Therapies to restore the enzyme’s function in the brain hold great promise for treating the neurological implications. Thus, we developed blood-brain barrier penetrant therapeutic molecules by fusing transferrin receptor-binding moieties to β-glucocerebrosidase (referred to as GCase-BS).