Poupon-Bejuit, L., Hughes, M. P., Liu, W., Geard, A., Faour-Slika, N., Whaler, S., . . . Rahim, A. A. (2022). A GLP1 receptor agonist diabetes drug ameliorates neurodegeneration in a mouse model of infantile neurometabolic disease. Scientific Reports, 12 (1), 13825. doi:10.1038/s41598-022-17338-1

Massaro, G., Colic, I., Whiteley, Z., Geard, A. F., Craig, D. Q. M., & Rahim, A. A. (2022). Evaluation of PEI-Based Viral Vector Production for Research-Grade Vector Manufacturing.

Ng, J., Barral, S., Barrigon, C. D. L. F., Lignani, G., Erdem, F., Wallings, R., . . . Kurian, M. A. (2021). Gene therapy for Dopamine transporter deficiency syndrome: Infantile Parkinsonism-dystonia.

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

Ng, J., Barral, S., De La Fuente Barrigon, C., Lignani, G., Erdem, F. A., Wallings, R., . . . Kurian, M. A. (2021). Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism. Science Translational Medicine, 13 (594). doi:10.1126/scitranslmed.aaw1564

Victor, S., Rocha-Ferreira, E., Rahim, A., Hagberg, H., & Edwards, D. (2021). New possibilities for neuroprotection in neonatal hypoxic-ischemic encephalopathy. European Journal of Pediatrics. doi:10.1007/s00431-021-04320-8

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. Biochimica et Biophysica Acta - Molecular Basis of Disease, 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 (vol 143, pg 1964, 2020). BRAIN, 143 (12), ARTN e106. doi:10.1093/brain/awaa272