Bridging patients and researchers to advance research in rare diseases

About Dr. Ryszard Kole
Dr. Kole is a pioneer in the use of oligonucleotides for the modulation of splicing. He received his Ph.D. from the Institute of Biochemistry and Biophysics of the Polish Academy of Sciences. As a postdoctoral fellow in the laboratory of Dr. Sidney Altman at Yale University, he provided early evidence that an RNA component is essential for the activity of a tRNA processing enzyme, RNase P. Subsequent discovery that RNase P is a ribozyme was recognized by a Nobel Prize for Dr. Altman. After joining the Department of Pharmacology at the University of North Carolina at Chapel Hill as a faculty member, Dr. Kole’s work led to a discovery that splicing provides a novel target for gene based therapies of numerous disorders including cancer, genetic diseases and metabolic disorders such as obesity. He is currently participating in developing this technology into treatment for DMD at AVI Biopharma.