Marrone, Lara

Isogenic FUS-eGFP iPSC Reporter Lines Enable Quantification of FUS Stress Granule Pathology that Is Rescued by Drugs Inducing Autophagy

Marrone, L., Poser, I., Casci, I., Japtok, J., Reinhardt, P., Janosch, A., Andree, C., Lee, H. O., Moebius, C., Koerner, E., et al. (2018) · Stem Cell Reports

Amyotrophic Lateral Sclerosis ResearchPluripotent Stem Cells Research

Cited by 104

Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders

Marrone, L., Marchi, P. M., & Azzouz, M. (2022) · Expert Opinion on Biological Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 56

Delivery of therapeutic AAV9 vectors via cisterna magna to treat neurological disorders

Marchi, P. M., Marrone, L., & Azzouz, M. (2022) · Trends in Molecular Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 21