Final Program - American Society of Gene & Cell Therapy

Program Schedule, Friday, May 17, 2013
Scientific Symposium 311
9:45 am - 11:45 am
ROOM: BALLROOM B
Cell and Tissue Engineering for Musculoskeletal Disease
CO-CHAIRS: Alan J. Nixon, DVM, MS and Bing Wang, MD, PhD
SPEAKERS
Rita Perlingeiro, PhD
From iPS cells to Skeletal Muscle Precursors: Potential for Gene Correction and Therapeutic Application
A major obstacle in the application of cell-based therapies for the treatment of neuromuscular disorders is obtaining the
appropriate number of stem/progenitor cells to produce effective engraftment. The use of pluripotent embryonic stem (ES) or
induced pluripotent stem (iPS) cells could overcome this hurdle. We have developed a method to eficiently generate skeletal
myogenic progenitors from pluripotent stem cells through Pax3 or Pax7 induction. We have applied this method to mouse ES
and iPS cells, and more recently to human ES and iPS cells. In each case, Pax7-induced cells engraft extensively and promote
improved contractility in transplanted dystrophic muscles (using a mouse model for Duchenne Muscular Dystrophy). In addition
to generating new myoibers, the transplanted cells also seeded the muscle satellite cell compartment. Accordingly, in long-term
cohorts, the frequency of human-speciic dystrophin+ ibers declined only modestly at one year post-transplant, compared to one
month. Based on these indings, we are now focusing on the genetic correction of iPS cells encoding mutations in the dystrophin
gene. Such studies are crucial for the future therapeutic application of iPS-derived myogenic progenitors in the autologous setting
for DMD. These more recent studies will also be discussed.
Rocky S. Tuan, PhD
Regenerative Applications of Adult Stem Cells
Farshid Guilak, PhD
Engineering Functional Tissue Replacements to Treat Osteoarthritis
Osteoarthritis is painful and debilitating joint disease that is characterized by pain and degenerative changes in the articular
cartilage and other joint tissues. While a number of techniques have been developed over the years for the treatment of small
cartilage defects, there have been few attempts at tissue-engineered therapies for end-stage osteoarthritis. Some of the major
considerations for this approach include the identiication and characterization of an abundant and accessible cell source as well as
the design of biologically and mechanically functional scaffolds that can withstand joint loading. Here we describe the engineering
of large, anatomically-shaped cartilage constructs using different stem cell sources (adipose stem cells, mesenchymal stem cells,
or induced pluripotent stem cells), combined with mechanically functional cell-instructive scaffolds based on three-dimensional
weaving of biocompatible ibers. As each iber is selected individually, this textile processing technique allows site-speciic
functionalization of proteins or virus to facilitate the formation of complex inhomogenous tissues from a single cell source.
Brian Johnstone, PhD
Stem Cells for Cartilage Repair
Scientific Symposium 312
9:45 am - 11:45 am
ROOM: 150 DEFG
Gene Therapy and Regenerative Medicine for Ocular and Metabolic Diseases
CO-CHAIRS: Alberto Auricchio, MD and Thierry C. VandenDriessche, PhD
SPEAKERS
Friday, May 17, 2013
Ian Constable, AO
Development of AAV-sFLT-1 for the Treatment of Wet Age-related Macular Degeneration
The eye is an attractive organ in which to develop gene therapy. There is relative ease of access, a benign immunological response
and the availability of high resolution non-invasive structural and functional tests. The safety and possible eficacy of Adenoassociated
Viral (AAV) gene therapy is now well established in young humans with RPE-65 defective retinal blindness. We have
adopted similar techniques to treat wet macular degeneration by subretinal injection of AAV-sFLT-1 to up-regulate production of
sFLT-1, a competitive analogue of the soluble Vascular Endothelial Growth Factor receptor.
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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