Therapeutic Potential of RNAi Through Endocytotic Methods
Year: 2016
Type: dissertation
Abstract: The ability to manipulate gene expression promises to be an important tool for the management of infectious diseases and genetic disorders. However, a major limitation to effective delivery of therapeutic RNA to living cells is the cellular toxicity of conventional techniques. Team PANACEA’s research objective was to create new reagents based on a novel small-molecule delivery system that uses a modular recombinant protein vehicle consisting of a specific ligand coupled to a Hepatitis B Virus-derived RNA binding domain (HBV-RBD). Two such recombinant delivery proteins were developed: one composed of Interleukin-8, the other consisting of the Machupo Virus GP1 protein. The ability of these proteins to deliver RNA to cells were then tested. The non-toxic nature of this technology has the potential to overcome limitations of current methods and could provide a platform for the expansion of personalized medicine.
DOI: 10.13016/m2nr2w
Authors: Dong, Joe, Giromini, Christopher, Han, Woojin, Hatten, Sonja, Kim, Ki, Pahlavan, Autusa, Patel, Rajan, Ufot, Ani, Young, LeAnne
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W2411255867