Funded Research

Published Research Funded by Rare Trait Hope Fund (United States)

– Banning, A., Gülec, C., Rouvinen, J. et al. Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria. Sci Rep 6, 37583 (2016). https://doi.org/10.1038/srep37583

– Banning, A.; König, J.F.; Gray, S.J.; Tikkanen, R. Functional Analysis of the Ser149/Thr149 Variants of Human Aspartylglucosaminidase and Optimization of the Coding Sequence for Protein Production. Int. J. Mol. Sci. 2017, 18, 706. https://doi.org/10.3390/ijms18040706

– Banning, A.; König, J.F.; Gray, S.J.; Tikkanen, R. Functional Analysis of the Ser149/Thr149 Variants of Human Aspartylglucosaminidase and Optimization of the Coding Sequence for Protein Production. Int. J. Mol. Sci. 2017, 18, 706. https://doi.org/10.3390/ijms18040706

– Antje Banning, Manuel Schiff, Ritva Tikkanen (2018), Amlexanox provides a potential therapy for nonsense mutations in the lysosomal storage disorder Aspartylglucosaminuria. Biochimica et Biophysica Acta, 1864:668-675. https://doi.org/10.1016/j.bbadis.2017.12.014

– Goodspeed, K., Harder, L., Hughes, S., Conger, D., Taravella, J., Gray, S.J. and Minassian, B. (2018), Optical coherence tomography features in brothers with aspartylglucosaminuria. Ann Clin Transl Neurol, 5: 1622-1626. https://doi.org/10.1002/acn3.672

– Xin Chen, Sarah Snanoudj-Verber, Laura Pollard, Yuhui Hu, Sara S. Cathey, Ritva Tikkanen, Steven J. Gray, Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation, Molecular Therapy, Volume 29, Issue 3, 2021, Pages 989-1000, https://doi.org/10.1016/j.ymthe.2020.11.012.