Article
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells.
Cell stem cell - 4 Jun 2020
Nagpal Neha, Wang Jianing, Zeng Jing, Lo Emily, Moon Diane H, Luk Kevin, Braun Roman O, Burroughs Lauri M, Keel Sioban B, Reilly Christopher, Lindsley R Coleman, Wolfe Scot A, Tai Albert K, Cahan Patrick, Bauer Daniel E, Fong Yick W, Agarwal Suneet
Abstract excerpt
Genetic lesions that reduce telomerase activity inhibit stem cell replication and cause a range of incurable diseases, including dyskeratosis congenita (DC) and pulmonary fibrosis (PF). Modalities to restore telomerase in stem cells throughout the body remain unclear. Here, we describe small-molecule PAPD5 inhibitors that demonstrate telomere restoration in vitro, in stem cell models, and in vivo. PAPD5 is a...
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