Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells
Abstract
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 non-canonical polymerase that...
Paper Details
Title
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells
Published Date
Jun 1, 2020
Journal
Volume
26
Issue
6
Pages
896 - 909.e8
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