research projects

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​Genetic Delivery of RNA Therapeutics for the Treatment for GBM

1. microRNA mimicry

• To deliver anti-GBM RNA interference.
• To design, synthesis and testing the genetic delivery of a miRNA gene architecture using microRNA gene cluster
• To lower the stability and reduce translation of the EGFR transcript in GBM.

2. Anti-Sense RNA 

• To block splicing and generate alternative therapeutic isoforms. This Aim has two parts, a) Design of the therapy vector platform and b) Synthesis of a Mini-Reporter Construct to Test Gene Transfer of RNA Therapeutics. 2a) Design, synthesis and initial testing of a multifunctional RNA anti-sense gene transfer strategy to block splicing and induce alternative and therapeutic isoforms of TKRs, such as hepatocyte growth factor receptor (HGFR) and VEGFR2 expression in GBM. 2b) To develop a mini-reporter system to test and optimize intron and transcript specific target, beginning with exon 9 through exon 10 of VEGFR2 and to include other regions of VEGFR2 and other TKRs (including HGFR).

3. Pre-Trans-Splicing

• Delivery of a Pre-trans-splicing RNA (PTR) therapy to generate alternative therapeutic isoforms with greater therapeutic potential. ​

5. Antibody Delivery

• AAV delivery of genes that encode anti-GBM antibodies directed against EGFR, VEGF and CXCR/ SDF-1a axis in GBM. 5a) anti-EGFR antibody blocking proliferation. 5b) anti-VEGF antibody blocking angiogenesis. 5c) anti-SDF-1a blocking hypoxia-induced tumor invasion.

4. Exosome Signal for RNA Delivery 

• Exosomal RNA “zipcode” signal to extend RNA therapy to tumor micro-environment​

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Environmental DNA Project                                             

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Vaccines Against Small Molecule Drugs Project

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