Direct Administration and Gene Modulation Using Antisense Oligonucleotides Within the CNS.

Abstract

Besides his vast contribution to the opioid receptor studies, Dr. G. W. Pasternak was among the early pioneers in the antisense oligonucleotide (ASO) field at the time when the crucial in vivo studies using ASO-mediated gene knockdown in the CNS were still impeded by the ASO's inability to cross the blood-brain barrier. This changed at the start of 1990s, when administration of oligonucleotides through intracerebroventricular or, later, intrathecal injection was undertaken at Cornell University Medical College and further developed in close collaboration with Pasternak lab. These early studies eventually led to the practical realization of the significant therapeutic potential of ASO-based drugs we see today.