Sabnis, Staci

A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

Sabnis, S., Kumarasinghe, E. S., Salerno, T., Mihai, C., Ketova, T., Senn, J. J., Lynn, A., Bulychev, A., McFadyen, I., Chan, J., et al. (2018) · Molecular Therapy

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 820

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

An, D., Schneller, J. L., Frassetto, A., Liang, S., Zhu, X., Park, J.-S., Theisen, M., Hong, S.-J., Zhou, J., Rajendran, R., et al. (2017) · Cell Reports

RNA Interference and Gene DeliveryMetabolism and Genetic Disorders

Cited by 263

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

Jiang, L., Berraondo, P., Jericó, D., Guey, L. T., Sampedro, A., Frassetto, A., Benenato, K. E., Burke, K., Santamaría, E., Alegre, M., et al. (2018) · Nature Medicine

Porphyrin Metabolism and DisordersRNA Interference and Gene Delivery

Cited by 181

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

mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency

Cao, J., An, D., Galduroz, M., Zhuo, J., Liang, S., Eybye, M., Frassetto, A., Kuroda, E., Funahashi, A., Santana, J., et al. (2019) · Molecular Therapy

RNA Interference and Gene DeliveryMetabolism and Genetic Disorders

Cited by 70