Judy Lieberman, MD, PhD, on Developing siRNA Therapies That Target Specific Cell Types


The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed past, present, and potential future applications of siRNA in the treatment of various diseases.

“I was involved in [this] field from the beginning. I was the first person who showed you could use siRNAs in an animal model to treat disease and also to develop targeted RNA therapy where you could design RNAs that got into a specific cell type like a T-cell or a cancer cell and knock down one specific gene, for therapy.”

Currently, there are 5 FDA-approved therapeutics based on small interfering RNA (siRNA), a type of RNA that can regulate expression of a specific gene. Although nonclinical research indicates that siRNA-based therapies could potentially be used to target a wide range of cell types, all currently approved products primarily impact the cells of the liver. As such, until methods of effectively targeting siRNA to other cell types are established, the therapeutic applications of siRNA are limited.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, is currently investigating potential methods to overcome this limitation so that the siRNA modality may one day be able to be used for the treatment of cancers, the prevention of HIV transmission, and potentially other applications as well. Lieberman, who notably was the first person to show in animal models that siRNA can be used to treat disease, presented on research in the field of siRNA therapeutics at the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024.

Shortly after her talk, CGTLive® sat down with Lieberman to learn more about her research in siRNA therapeutics. Lieberman went over the history siRNA therapy research, the present state of approved siRNA therapies, and her nonclinical research on methods of directly targeting siRNA therapies to specific cell types. She spoke about 2 particular methods she has explored in her research: the use of antibody fragments fused with protamine and the use of aptamer-conjugated RNAs.

Click here for more coverage of Tandem 2024.

1. Lieberman J. The silent treatment: siRNA therapeutics. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas.
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