Leaf Huang, Ph.D., is the Fred Eshelman Distinguished Professor, Division of Molecular Pharmaceutics in the Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, U.S.A. Dr. Huang’s research has been in the area of gene therapy and targeted drug delivery. He has pioneered the liposome nonviral vector and has designed and manufactured the cationic lipid vector for the first nonviral clinical trial in 1992. His current work centers on nanoparticle vectors for gene transfer in tumors and the liver. He also continues research in establishing a ligand-targeted delivery system for cDNA, mRNA, siRNA, proteins, and peptides for tumor growth inhibition and for vaccines in treating cancer and infectious diseases. He has authored or coauthored more than 320 peer-reviewed papers and more than 130 reviews and book chapters. The H-index of his publications is 76. He is also the inventor or coinventor of 16 U.S. and foreign patents. In 2004, he received the Alec D. Bangham, MD, FRS Achievement Award, which is the highest honor in liposome research. Dr. Huang has also cofounded five biotech start-ups in the past.
It has been predicted that, by 2014, seven out of the top ten drugs will be biopharmaceuticals, mainly recombinant proteins including monoclonal antibodies. However, most if not all existing protein drugs have their targets at or outside the cell surface. This is because macromolecules, including proteins, do not easily penetrate into the cells. I focus my research on intracellular delivery of genes (including cDNA, mRNA, miRNA, siRNA, and antisense oligonucleotide), proteins, peptides, and small drugs for various targets including tumor, liver, lung, and dendritic cells in the lymph nodes. Just imagine, if active proteins can be efficiently delivered to their intracellular compartment (cytoplasm, nucleus, mitochondrion, endoplasmic reticulum, etc.), there will be many new molecular targets available for drug development. Our recent work (Biomaterials, 2012) in delivering cytochrome C to induce apoptosis of tumor cells in a xenograft model is a good example. We have also succeeded in delivering a nonapeptide to block the cytoplasmic kinase domain of the EGF receptor kinase in tumor cells (J. Controlled Release, 2011). Several different membrane/core nanoparticles have been designed by us using the principles of self-assembly chemistry and physics. These nanoparticles evade the reticuloendothelial system and deliver and release their cargos to the intracellular sites of action. If elected as a member of the Board of Scientific Advisors, I will advise the members of the Controlled Release Society on the importance and the practice of intracellular delivery of macromolecules.