Article
Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct.
Nature communications - 12 Apr 2023
Gehrlein Alexandra, Udayar Vinod, Anastasi Nadia, Morella Martino L, Ruf Iris, Brugger Doris, von der Mark Sophia, Thoma Ralf, Rufer Arne, Heer Dominik, Pfahler Nina, Jochner Anton, Niewoehner Jens, Wolf Luise, Fueth Matthias, Ebeling Martin, Villaseñor Roberto, Zhu Yanping, Deen Matthew C, Shan Xiaoyang, Ehsaei Zahra, Taylor Verdon, Sidransky Ellen, Vocadlo David J, Freskgård Per-Ola, Jagasia Ravi
Abstract excerpt
Mutations 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...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.