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Research Overview

The Hirsch Lab works on Adeno-Associated Virus (AAV) biology and the development AAV Gene Therapeutics.

 

  • Synthetic Inverted Terminal Repeats…We have a panel of rationally designed ITRs that alter nearly all aspects of AAV vector biology including production, transduction efficiency, and the cellular responses to the vector
  • AAV vectors and “Helper” viruses…The evolved relationship of AAV with other viruses is down right intriguing, and offers clues about AAV vector optimization towards safer more efficient therapeutic strategies
  • Regulation of Transgenic Gene Expression…The community needed this one yesterday
  • Anterior Eye Therapeutics…AAV vector applications in the anterior eye have been largely overlooked, we are addressing that deficit towards improved vision in patients with a variety of ocular diseases
  • Muscle Therapeutics…In particular, we are investigating a potential AAV therapeutic for the treatment of dysferlinopathy