Supported Research

A small molecule approach to overcome replication dysfunction in FA

2020 | Univ. of Massachusetts & The Children’s Hospital of Philadelphia | Research Grant

Amount Funded: $250,000

The goal of this project is to identify drugs that improve DNA integrity and bone marrow stem cell function in FA. The Cantor lab has uncovered defects in so-called DNA replication that lead to small gaps and compromise cell turnover and fitness of FA cells. Striking similarities with the data generated in FA mice by the Kurre lab suggest that this mechanism may be the root of bone marrow failure (BMF) in FA patients by inadvertently activating a safety switch that prevents blood stem cell formation. For this purpose, we have designed an experimental platform that enables us to identify drugs that activate pathways that suppress gaps and in turn improve DNA replication, cell fitness, and FA blood stem cell function.

Researchers: Peter Kurre, Sharon Cantor