Final Program - American Society of Gene & Cell Therapy

Program Schedule, Friday, May 17, 2013
Scientific Symposium 316
9:45 am - 11:45 am
ROOM: 150 ABC
Partially supported by the Juvenile Diabetes Research Foundation.
Gene and Cell Therapy for Diabetes
CO-CHAIRS: Nicola Brunetti-Pierri, MD and Lawrence C. Chan, MD
SPEAKERS
H. Teresa Ku, PhD
Pancreatic Stem and Progenitor Cells
Adult pancreatic stem and progenitor cells are potential unlimited sources of beta cells, however their existence is highly
controversial. To address this issue, we report here the establishment of two in vitro pancreatic colony assays that enable
quantitative measurement of self-renewal and differentiation at a single cell level, which is required to deinitively demonstrate
progenitor cell activities. We ind that single pancreatic colony-forming cells are capable of self-renewal in the presence of Matrigel
and a Wnt signaling agonist, and can differentiate into ductal-like cells (in the presence of Matrigel) as well as endocrine and acinar
lineage cells (in the presence of an unique laminin hydrogel) in culture. These results demonstrate robust pancreatic progenitor cell
activities in vitro. Our results call for further investigation of progenitor cells in the adult pancreas in vivo, a research activity that
has largely ceased after a series of recent publications reporting negative indings.
Lawrence C. Chan, MD
Gene Therapy to Reverse Autoimmune Type 1 Diabetes
Type 1 diabetes (T1D) happens when most of the body’s pancreatic beta cells are destroyed by autoimmunity. Therefore, to reverse
diabetes in T1D patients, we need to [1] re-establish suficient beta cell mass, and [2] neutralize the effects of autoimmunity. Our
laboratory has developed a strategy that couples gene therapy-mediated induction of new beta cell formation in the liver with an
immunomodulatory regimen that protects the neo-beta cells from autoimmune destruction. This strategy successfully reverses
diabetes in an autoimmune model in rodents; the concept of therapeutic hepatic islet induction may also work in nonhuman
primates.
Andrea Ballabio, MD
Rescue of Fat Diet-induced Obesity and Diabetes by TFEB-mediated Regulation of Autophagy and Lipid
Catabolism
The lysosomal-autophagic pathway is activated by starvation and plays an important role in both cellular clearance and lipid
catabolism. However, the transcriptional regulation of this pathway in response to metabolic cues is currently uncharacterized.
Here we show that the transcription factor EB (TFEB), a master regulator of lysosomal biogenesis and autophagy, is induced by
starvation through an autoregulatory feedback loop and exerts a global transcriptional control on lipid catabolism via PGC1α and
PPARα. Thus, during starvation a transcriptional mechanism links the autophagic pathway to cellular energy metabolism. Viral
delivery of TFEB to the liver rescued obesity, diabetes and the metabolic syndrome in both diet-induced and genetic mouse models
of obesity, suggesting a novel therapeutic strategy for disorders of lipid metabolism.
Friday, May 17, 2013
Markus Grompe, MD
Reprogramming Gall Bladder to make Insulin
Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter)
11:45 am - 1:00 pm
Oral Abstract Session 320
1:00 pm - 3:00 pm
ROOM: BALLROOM A
DNA Vectorology & Gene Targeting
CO-CHAIRS: Matthew Hirsch, PhD and Carol H. Miao, PhD
(Abstracts 266 through 273; see page 108)
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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