Heartlein, Michael W.

Optimization of a degradable polymer–lipid nanoparticle for potent systemic delivery of mRNA to the lung endothelium and immune cells

Kaczmarek, J. C., Kauffman, K. J., Fenton, O. S., Sadtler, K., Patel, A. K., Heartlein, M. W., DeRosa, F., & Anderson, D. G. (2018) · Nano Letters

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

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mRNA Delivery for Therapeutic Anti-HER2 Antibody Expression In Vivo

Rybakova, Y., Kowalski, P. S., Huang, Y., Gonzalez, J. T., Heartlein, M. W., DeRosa, F., Delcassian, D., & Anderson, D. G. (2019) · Molecular Therapy

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 218

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

DeRosa, F., Guild, B., Karve, S., Smith, L., Love, K., Dorkin, J. R., Kauffman, K. J., Zhang, J., Yahalom, B., Anderson, D. G., et al. (2016) · Gene Therapy

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 159

CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

Calias, P., Papisov, M., Pan, J., Savioli, N., Belov, V., Huang, Y., Lotterhand, J., Alessandrini, M., Liu, N., Fischman, A. J., et al. (2012) · PLoS ONE

Lysosomal Storage Disorders ResearchCerebrospinal fluid and hydrocephalus

Cited by 133

Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy

DeRosa, F., Smith, L., Shen, Y., Huang, Y., Pan, J., Xie, H., Yahalom, B., & Heartlein, M. W. (2019) · Molecular Therapy

Lysosomal Storage Disorders ResearchRNA Interference and Gene Delivery

Cited by 111

Polymer–Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs

Kaczmarek, J. C., Patel, A. K., Kauffman, K. J., Fenton, O. S., Webber, M. J., Heartlein, M. W., DeRosa, F., & Anderson, D. G. (2016) · Angewandte Chemie

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 60