Jones, D. J., Soundararajan, D., Taylor, N. K., Aimiuwu, O. V., Mathkar, P., Shore, A., Teoh, J. J., Wang, W., Sands, T. T., Weston, M. C., Harper, S. Q., & Frankel, W. N. (2024). Effective knockdown-replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2024.08.009

Occelli, L. M., Zobel, L., Stoddard, J., Wagner, J., Pasmanter, N., Querubin, J., Renner, L. M., Reynaga, R., Winkler, P. A., Sun, K., Marinho, L. F. L. P., O’Riordan, C. R., Frederick, A., Lauer, A., Tsang, S. H., Hauswirth, W. W., McGill, T. J., Neuringer, M., Michalakis, S., & Petersen-Jones, S. M. (2023). Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa. Molecular Therapy, 31(7), 2028–2041. https://doi.org/10.1016/j.ymthe.2023.04.005

Selles, M. C., Fortuna, J. T. S., Cercato, M. C., Santos, L. E., Domett, L., Bitencourt, A. L. B., Carraro, M. F., Souza, A. S., Janickova, H., Azevedo, C. V., Campos, H. C., de Souza, J. M., Alves-Leon, S., Prado, V. F., Prado, M. A. M., Epstein, A. L., Salvetti, A., Longo, B. M., Arancio, O., … Ferreira, S. T. (2023). AAV-mediated neuronal expression of an scFv antibody selective for Aβ oligomers protects synapses and rescues memory in Alzheimer models. Molecular Therapy, 31(2), 409–419. https://doi.org/10.1016/j.ymthe.2022.11.002

Pepe, G., Capocci, L., Marracino, F., Realini, N., Lenzi, P., Martinello, K., Bovier, T. F., Bichell, T. J., Scarselli, P., Di Cicco, C., Bowman, A. B., Digilio, F. A., Fucile, S., Fornai, F., Armirotti, A., Parlato, R., Di Pardo, A., & Maglione, V. (2023). Treatment with THI, an inhibitor of sphingosine-1-phosphate lyase, modulates glycosphingolipid metabolism and results therapeutically effective in experimental models of Huntington’s disease. Molecular Therapy, 31(1), 282–299. https://doi.org/10.1016/j.ymthe.2022.09.004

Wu, W.-H., Tsai, Y.-T., Huang, I.-W., Cheng, C.-H., Hsu, C.-W., Cui, X., Ryu, J., Quinn, P. M. J., Caruso, S. M., Lin, C.-S., & Tsang, S. H. (2022). CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa. Molecular Therapy, 30(4), 1407–1420. https://doi.org/10.1016/j.ymthe.2022.02.010