Joseph R. Dorkin

TL;DR: An introduction to the biological challenges that siRNA delivery materials aim to overcome is provided, as well as a discussion of the way that the most effective and clinically advanced classes of si RNA delivery systems are designed to surmount these challenges.

TL;DR: The delivery strategy is applied to a mouse model of human hereditary tyrosinemia and it is shown that the treatment generated fumarylacetoacetate hydrolase (Fah)-positive hepatocytes by correcting the causative Fah-splicing mutation and rescued disease symptoms such as weight loss and liver damage.

TL;DR: It is demonstrated that LNP composition alone can be used to modulate the site of protein induction in vivo, highlighting the critical importance of designing and synthesizing new nanomaterials for nucleic acid delivery.

TL;DR: In this paper, nanoparticles formulated with lipids and lipid-like materials were developed for delivery of two separate mRNA transcripts encoding either human erythropoietin (hEPO) or factor IX (hFIX) protein.

TL;DR: Therapeutic value was established within a hemophilia B ( FIX knockout (KO) mouse model by demonstrating a marked reduction in Hct loss following injury (incision) to FIX KO mice, and the capacity of producing EPO in non-human primates via delivery of formulated mRNA was demonstrated as elevated EPO protein levels were observed over a 72-h time course.