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.
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.

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.