Nucleic Acid (NA) based therapeutics are poised to disrupt modern medicine and augment traditional pharmaceutics in a meaningful way. However, a key challenge to advancing NA therapies into the clinical setting and on to the market is the safe and effective delivery to the target tissue and cell. Lipid Nanoparticles (LNP) have been extensively investigated and are currently the most advanced vector for the delivery of NA drugs, as evidenced by the approval of Onpattro for treatment of Amyloidosis in the US and EU in 2018. This article provides a comprehensive review of the state-of-the-art for LNP technology. We discuss key advances in the design and development of LNP, leading to a broad range of therapeutic applications. Finally, the current status of this technology in clinical trials and its future prospects are discussed.
Publication - Abstract
Dec 21, 2017
Gastroenterology
Cytokinesis can fail during normal postnatal liver development, leading to polyploid hepatocytes. We investigated whether inhibiting cytokinesis in the liver slows tumor growth without compromising the health of normal hepatocytes. We inhibited cyt...
Publication - Abstract
Apr 03, 2016
Nucleic Acids
The androgen receptor plays a critical role in the progression of prostate cancer. Here, we describe targeting the prostate-specific membrane antigen using a lipid nanoparticle (LNP) formulation containing small interfering RNA (siRNA) designed to ...