this combination of methods is to give investigators more powerful tools with which to alter how the genome is expressed in cells and silence disease-causing genes."
For example, Fliesler and his UB colleagues are conducting research on novel gene therapy applications to treat retinitis pigmentosa, a group of genetic eye conditions that can lead to incurable blindness and which Fliesler says underscores the importance of genomic research.
In retinitis pigmentosa, he says, there are well over a hundred known mutations in the gene that codes for the visual pigment rhodopsin alone, and there are dominant and recessive forms of the disease.
"If it was possible to just disable the disease-causing allele (one member of a pair of genes) early in development, then you'd get a normal individual," Fliesler says.
Plenary lectures will be given by Larry A. Sklar, PhD, of the University of New Mexico, John S. Lazo, PhD, of the University of Pittsburgh, Bryan Roth, MD, PhD, of the University of North Carolina at Chapel Hill and Menghang Xia, PhD, of the National Institutes of Health.
Topics of other talks will include:
- Advances in genomic techniques that have allowed scientists to dissect how the cell responds to changes in the environment by modulating access to information encoded in the genome. The talk, by Michael J. Buck, PhD, UB assistant professor of biochemistry, will focus on a master regulator essential for cellular stress response and how it controls access to the genomic information.
- challenges in developing RNA drugs and ways that UB scientists and others are working to overcome them. Development of RNA drugs as novel gene-based therapies for retinitis pigmentosa and other retinal degenerations is a primary focus of the research program of Jack M. Sullivan, MD, PhD, UB associate professor of ophthalmology, who will discuss an experimental platform his group has developed to rapidly screen large sets of candidate RNA drugs to i
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