David Schaffer, PhD


Our research program focuses on the related and increasingly integrated fields of gene therapy and stem cell biology. Specifically, we have developed directed evolution approaches to engineer novel viral vector variants, particularly adeno-associated virus and lentivirus, with enhanced gene delivery properties for therapeutic applications. For example, we have evolved new viral variants with altered receptor binding specificity, enhanced infection of non-permissive cells, targeted gene delivery in vitro and in vivo, improved the ability to evade pre-existing immunity, and developed safer integration into the human genome.

Furthermore, we are exploring the therapeutic potential of these variants to treat disease in the central nervous system, retina, and lung. In parallel, we apply stem cell biology and bioengineering approaches to control the behavior of neural and human embryonic stem cells and thereby enhance the efficiency of their differentiation into therapeutically valuable cell types for cell replacement therapies. It is our hope that gene and stem cell therapies, both individually and in combination, will progressively lead to new cures for numerous devastating human diseases.


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22nd Annual Meeting
April 29 – May 2 | Washington D.C.