PROJECT SUMMARY Human genome project evolved with the promise of offering genetic modification as a tool to treatsevere and debilitating diseases. Gene therapy has emerged as an important tool to modulate geneexpression for treating various diseases. Development of gene medicines requires the rational nucleicacid drug design with unparalleled specificity and rapidity of development. Despite of manyadvantages, only two nucleic acid-based therapies have been approved: fomivirsen (Vitravene, anantisense phosphorothioate oligonucleotides) and pegaptanib (Macugen, an aptamer) for localintraocular therapy highlighting the challenges in systemic therapy of nucleic acids. Inefficient cellularuptake, poor plasma stability, intracellular trafficking and immunogenicity are some of the key hurdlesin the translation of nucleic acids into therapeutics. These limitations can be potentially overcomethrough 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 stilla lack of platforms where scientists from pharma, biotech, academia and regulatory agencies canmeet to share their expertise and discuss strategies to overcome challenges related to systemicdelivery of nucleic acids and product development. Nucleic acid-based therapeutics requires diversedisciplines including biochemistry and molecular biology, material science, drug delivery,pharmacokinetics, genomics, bioengineering and informatics. This two day symposium will provideopportunity to the key players working on different aspects of gene medicines to share theirknowledge and thoughts on further improvement. The symposium will have interdisciplinary focuswith participation of scientists from diverse institutions and scientific disciplines toshare/discuss/debate topics related to challenges of gene therapy and potential approaches for itsrapid clinical development. Financial support provided for this symposium will allow the participationof trainees/young investigators as well as a diverse group of individuals including those representingminorities and traditionally underrepresented communities in science careers. In addition, registrationfee waivers and travel awards will be provided to encourage the participation by minorities andunderrepresented communities. There will also be oral and poster presentations from selectedgraduate students and young investigators.
|Effective start/end date||7/15/16 → 6/30/17|
- National Institutes of Health: $10,000.00
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