Key points from article :
Team described PAPD5 inhibitors that demonstrate telomere restoration.
Demonstrated in vitro, in stem cell (SC) models, and in vivo.
And have identified BCH001, a specific PAPD5 inhibitor.
Human blood SCs engineered to carry dyskeratosis congenita (DC) -causing PARN mutations.
Xenotransplanted into immunodeficient mice, and orally treated with repurposed PAPD5 inhibitor.
Findings: It rescued TERC 3′ end maturation and telomere length.
This research may also have utility for the treatment of other age-related diseases.
Published in Cell Stem Cell.