The long view

While most of the activity in RNA-based therapeutics has focused on small RNAs that can dampen expression of disease-causing proteins, Jeannie Lee has built a career, and a company, on transcripts that are about 10 times as long and can be targeted to activate genes by relieving epigenetic silencing. She believes the long non-coding RNAs (lncRNAs) could add a new arm to drug development that could both compete with and complement traditional small molecules, as well as more recent modalities such as gene therapy and gene editing.

Lee, who is a professor of genetics and pathology at Harvard Medical School and Massachusetts General Hospital, discovered the importance of lncRNAs while studying X-linked diseases and the mechanisms underlying X-chromosome inactivation. That led to the identification of lncRNAs as important epigenetic modulators that control the expression of genes involved in a variety of diseases...