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Vector Innovation Discovery and Engineering (VIDE) Program

Our research centers on understanding adeno-associated virus (AAV) and utilizing our knowledge to develop gene therapy vector platforms to treat human diseases.

Capsid Discovery and Characterization

  • Using high-throughtput sequencing strategies, we aim to discover and characterize natural AAV capsid sequences for their potential use as gene therapy vectors.

 

 

Development of Quality Control Workflows for Gene Therapy Vectors

  • One understudied aspect of gene therapy vectorology is the relative frequency of heterogenic bi-products in the form of genomic impurities, truncations, and chimeric vector sequences.  We seek to develop NGS and bioinformatics tools to profile preclinical and research-grade rAAVs.

 

 

Immunology

  • The host immune response towards foreign antigens is the most challenging hurdle for gene therapy.  Nonetheless, Immunology remains the most complex area of study for understanding why vector-based gene therapies fall short of fully treating disease.
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Virology

  • To understand how vector-based gene therapies interact with the host/patient, address why these tools work the way they do, and further modulate vectors to enhance their biological properties, it is imperative to improve our knowledge of how wild-type viruses function and the mechanisms of their complex life-cycles.
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Vector Engineering

  • Vector safety and efficacy is controlled by multiple factors, ranging from the DNA sequence in the vector design, all the way to how the therapy is administered to the host/patient.  We aim to discover ways to improve upon vector design to ensure safe and potent treatments.