Therapeutic Potential of RNAi Through Endocytotic Methods
Therapeutic Potential of RNAi Through Endocytotic Methods
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Date
2016-05
Authors
Dong, Joe
Giromini, Christopher
Han, Woojin
Hatten, Sonja
Kim, Ki
Pahlavan, Autusa
Patel, Rajan
Ufot, Ani
Young, LeAnne
Advisor
Dinman, Jonathan
Citation
DRUM DOI
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.