Potential of Nanotechnology in Gene Therapy

Thursday, January 10, 2013
12:30 pm–2:00 pm 

Organized by the AAPS Nanotechnology focus group
Conducted by Leaf Huang, Ph.D., University of North Carolina
Moderated by Ram Mahato, Ph.D., University of Tennessee Health Science Center

About the Webinar

Gene therapy can be considered as an alternative to conventional chemotherapy or radiotherapy for the treatment of various pathological conditions. The concept of gene therapy is based on the transfer of genetic material into the specific cells of the body for either up-regulation or down-regulation of the target. However, the biggest bottleneck for effective gene therapy has been the delivery. Traditionally, viral vectors have been utilized for the delivery of the genetic material but the potential adverse effects such as immunogenicity and/or carcinogenicity has prompted researchers to look into alternatives such as lipid and polymeric material based non-viral vectors.

The objective of this webinar will be to provide basic understanding of concepts in gene therapy, discuss major barriers in developing delivery platforms for siRNA and/or miRNA, provide a road-map to effective gene delivery with emphasis on examples of non-viral based nanoparticle platform technologies. The webinar will be of special interest to scientists/students who are interested in this field and would like to learn about the potential of nanotechnology in gene therapy.

Webinar Agenda

  • Introduction to Gene Therapy
  • Roadmap of Effective Gene Delivery
  • Viral vs. Non-Viral Vectors for Gene Therapy
  • Potential of siRNA/miRNA Technology and Delivery Challenges
  • Specific Examples of Non-Viral Gene Delivery Platforms for siRNA
  • Concluding Remarks
About the Presenter

Leaf HuangLeaf 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. Huang’s research has been in the area of gene therapy and targeted drug delivery. He has pioneered the liposome nonviral vector and 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.

Huang 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. Huang has also cofounded five biotech start-ups in the past.