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Telomerase activity restored in vitro and in mice

27-Apr-2020

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.

Potential to support new therapies for dyskeratosis congenita, other age-related diseases

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Journal

Scientific journal covering stem cell biology.