Massaro, G., Geard, A. F., Liu, W., Coombe-Tennant, O., Waddington, S. N., Baruteau, J., . . . Rahim, A. A. (2021). Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development. Biomolecules, 11 (4), 611. doi:10.3390/biom11040611

Seker Yilmaz, B., Baruteau, J., Rahim, A. A., & Gissen, P. (2020). Clinical and Molecular Features of Early Infantile Niemann Pick Type C Disease. International Journal of Molecular Sciences, 21 (14), 5059. doi:10.3390/ijms21145059

Plotegher, N., Perocheau, D., Ferrazza, R., Massaro, G., Bhosale, G., Zambon, F., . . . Duchen, M. R. (2020). Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload (vol 17, pg 315, 2020). CELL DEATH AND DIFFERENTIATION, 27 (8), 2534. doi:10.1038/s41418-020-0525-0

Liu, W., Kleine-Holthaus, S. -. M., Herranz-Martin, S., Aristorena, M., Mole, S. E., Smith, A. J., . . . Rahim, A. A. (2020). Experimental gene therapies for the NCLs. Biochim Biophys Acta Mol Basis Dis, 165772. doi:10.1016/j.bbadis.2020.165772

Rahim, A. A., & Gissen, P. (2020). Gene therapy for global brain diseases: one small step for mice, one giant leap for humans. Brain, 143 (7), 1964-1966. doi:10.1093/brain/awaa189

Karda, R., Rahim, A. A., Wong, A. M. S., Suff, N., Diaz, J. A., Perocheau, D. P., . . . Waddington, S. N. (2020). Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors. Scientific Reports, 10 (1), 2121. doi:10.1038/s41598-020-59075-3

Poupon-Bejuit, L., Rocha-Ferreira, E., Thornton, C., Hagberg, H., & Rahim, A. A. (2020). Neuroprotective Effects of Diabetes Drugs for the Treatment of Neonatal Hypoxia-Ischemia Encephalopathy. Frontiers in Cellular Neuroscience, 14, 112. doi:10.3389/fncel.2020.00112

Rahim, A. A., Russell, C., & Mole, S. E. (2020). Special edition: The NCLs/batten disease. Biochim Biophys Acta Mol Basis Dis, 165824. doi:10.1016/j.bbadis.2020.165824

Massaro, G., Hughes, M. P., Whaler, S. M., Wallom, K. -. L., Priestman, D. A., Platt, F. M., . . . Rahim, A. A. (2020). Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes. Hum Mol Genet. doi:10.1093/hmg/ddz317

Ruiz, A. J., & Rahim, A. A. (2019). Bringing balance to the force–regulatable gene therapy for epilepsy. Gene Therapy. doi:10.1038/s41434-019-0083-6