Lyman L. Handy Colloquium

"Molecular Engineering of Stem Cell and Gene Therapies"Professor David Schaffer
Department of Chemical Engineering &The Helen Wills Neuroscience Institute
University of California at Berkeley & Lawrence Berkeley National LaboratoryAbstractNew molecular therapies based on gene delivery and stem cells have significant potential for tissue engineering and repair for numerous diseases. Before these approaches can succeed, however, a number of fundamental engineering challenges must be overcome, particularly in the nervous system, our tissue of interest.Gene therapy, the introduction of genetic material to the cells of a patient for therapeutic benefit, has the potential to directly translate the basic knowledge derived from the Human Genome Project into therapeutic benefit. However, the vehicles or vectors that deliver therapeutic genes still require engineering for enhanced efficiency and safety. Our efforts are focused on modifying these vehicles at the molecular level to overcome the common dilemma faced by all: they did not evolve in nature to perform the therapeutic endeavors we ask of them. We have developed novel approaches to engineer already promising gene delivery vehicles, the adeno-associated viral vector and lentiviral vector. Specifically, we are applying directed evolution approaches to overcome several challenges in vector performance, including its mass transport through tissue and cells and interactions with the immune system.Furthermore, gene therapy has enormous potential to synergize with stem cells to repair damaged tissue. Neural stem cells are present throughout the adult nervous system, but we must learn at a quantitative, molecular level the signaling mechanisms that control these cells before we can harness them. We have identified novel signaling factors that regulate neural stem cells and are investigating the mechanisms by which the cells process these signals into functional decisions. Specifically, we are exploring the hypothesis that cell switching between multiple steady states in gene regulation networks can serve as a general mechanism for the critical fate choices these cells must make as they differentiate into specific cell types, such as neurons. We hope that this basic knowledge can be applied, in combination with improved gene delivery vehicles, to regenerate neural tissue from the effects of neurodegenerative disorders such as Alzheimer's, Parkinson's, and Lou Gehrig's Diseases.Thursday, October 12, 2006
12:45 p.m.
OHE 122
Refreshments will be served after the seminar in the HED Lobby
The Scientific Community is cordially invited.

Location: Olin Hall of Engineering (OHE) - 122

Audiences: Everyone Is Invited

Contact: Petra Pearce