Human genome project evolved with the promise of offering genetic modification as a tool to treat severe and debilitating diseases. Gene therapy has emerged as an important tool to modulate gene expression for treating various diseases. Development of gene medicines requires the rational nucleic acid drug design with unparalleled specificity and rapidity of development. Despite of many advantages, only two nucleic acid-based therapies have been approved: fomivirsen (Vitravene, an antisense phosphorothioate oligonucleotides) and pegaptanib (Macugen, an aptamer) for local intraocular therapy highlighting the challenges in systemic therapy of nucleic acids. Inefficient cellular uptake, poor plasma stability, intracellular trafficking and immunogenicity are some of the key hurdles in the translation of nucleic acids into therapeutics. These limitations can be potentially overcome through the development and scale up of nanoparticulate based nucleic acid delivery technologies. Although innovative and exciting work is being led by the biotech industry and academics, there is still a lack of platforms where scientists from pharma, biotech, academia and regulatory agencies can meet to share their expertise and discuss strategies to overcome challenges related to systemic delivery of nucleic acids and product development. Nucleic acid-based therapeutics requires diverse disciplines including biochemistry and molecular biology, material science, drug delivery, pharmacokinetics, genomics, bioengineering and informatics. This two day symposium will provide opportunity to the key players working on different aspects of gene medicines to share their knowledge and thoughts on further improvement. The symposium will have interdisciplinary focus with participation of scientists from diverse institutions and scientific disciplines to share/discuss/debate topics related to challenges of gene therapy and potential approaches for its rapid clinical development. Financial support provided for this symposium will allow the participation of trainees/young investigators as well as a diverse group of individuals including those representing minorities and traditionally underrepresented communities in science careers. In addition, registration fee waivers and travel awards will be provided to encourage the participation by minorities and underrepresented communities. There will also be oral and poster presentations from selected graduate students and young investigators.

Public Health Relevance

Site-specific delivery of nucleic acids to target cells for efficient gene expression or silencing without any side effects remains a challenge. This two day symposium will provide unique platform to ensure participation of scientists of diverse expertise for discussion on ways of rapidly turning nucleic acids into effective gene medicines.

National Institute of Health (NIH)
National Institute of Biomedical Imaging and Bioengineering (NIBIB)
Conference (R13)
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Special Emphasis Panel (ZEB1-OSR-E (M1)S)
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Rampulla, David
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University of Nebraska Medical Center
Other Basic Sciences
Schools of Pharmacy
United States
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Chaudhary, Amit Kumar; Mahato, Ram I (2017) The third annual BRDS on research and development of nucleic acid-based nanomedicines. Drug Deliv Transl Res 7:188-193