Milton, Jaclyn

Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates

Zhu, X., Yin, L., Theisen, M., Zhuo, J., Siddiqui, S., Levy, B., Presnyak, V., Frassetto, A., Milton, J., Salerno, T., et al. (2019) · The American Journal of Human Genetics

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 165

Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia

An, D., Frassetto, A., Jacquinet, E., Eybye, M., Milano, J., DeAntonis, C., Nguyen, V., Laureano, R., Milton, J., Sabnis, S., et al. (2019) · EBioMedicine

RNA Interference and Gene DeliveryMetabolism and Genetic Disorders

Cited by 87