6月 10日 (二)
7:00 am - 6:00 pm Registration Open
7:30 am Breakfast Workshop
(Sponsorships Available)
8:15 Chairperson’s Remarks
8:20 A New Frontier in Gene Therapy
Eric Kmiec, Ph.D., Professor, Biology, University of Delaware/OrphageniX
Over the past seven years, we have pioneered the technique of gene repair wherein genetic mutations are corrected directly within context of the chromosome. This repair is facilitated by oligonucleotides , non-viral drug-like agents that does not induce an immune reaction or cause toxicity in humans. A number of genetic diseases including sickle cell anemia, spinal muscular atrophy and muscular dystrophy have been successfully treated in cell and animal models. Now, the focus is translating these results into a clinical trial.
8:50 Lasting Effects by Transient Gene Transfer: Epigenetics
Casey Case, Ph.D., Vice President Research, SanBio Inc.
Our cell therapy product is produced by transient transfection of Mesenchymal Stem Cells. The vector used encodes the Notch-1 IntraCellular Domain (NICD) a powerful regulator of devel-opmental cell fate. Transfection with this vector causes the cells to assume neuronal precursor-like properties and to lose the ability to differentiate down alternative paths. This beneficial effect persists long after the gene transfer vector is gone. We are exploring epigenetic mechanisms, such as CpG DNA methylation, to explain this phenomenon. These cells have shown beneficial results in models of stroke, Parkinson’s disease and spinal cord injury.
9:20 Sequence-Specific Modification of Genomic DNA by Oligonucleotides
Dieter Gruenert, Ph.D., Professor, Senior Scientist, Cell Biology, California Pacific Medical Center Research Institute
We have developed a strategy small fragment homologous replacement (SFHR) for modifying specific targets in the genomic DNA using small DNA fragments (SDF). Studies in hematopoie-tic stem cells, lymphoblasts, epithelial cells, and embryonic stem cells have shown SFHR-mediated modification. This approach has potential therapeutic applications as well as in the
development of transgenic animals.
9:50 Networking Coffee Break, Poster and Exhibit Viewing
10:45 Nucleic Acid Delivery and Gene Repair in the Eye
John M. Nickerson, Ph.D., Professor, Department of Ophthalmology, Emory University
Short single stranded oligonucleotides (ODNs) can be delivered into photoreceptor cells of the neural retina in vivo. We used a mouse strain bearing the retinal degeneration (rd1) lesion, a point mutation in a gene encoding the beta-subunit of cGMP phosphodiesterase (beta-PDE). Delivery of therapeutic ODNs to rd1 mouse eyes resulted in genomic DNA conversion from mutant to wild type sequence at a low but observable incidence. Correspondingly, observable beta-PDE immunoreactivity was detected. Rhodopsin immunopositive cells were detectable in the outer layers of the retina, suggesting that ODN-directed gene repair occurred in about 0.2% of cells.
11:15 Selected Brief Poster Presentation
