The DOI matches its Crossref record; 89 of the 89 references it cites were found in a registry or an index.
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells
Open accessAbstract
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 oligoadenylates and destabilizes telomerase RNA component (TERC). We identified BCH001, a specific PAPD5 inhibitor that restored telomerase activity and telomere length in DC patient induced pluripotent stem cells. When human blood stem cells engineered to carry DC-causing PARN mutations were xenotransplanted into immunodeficient mice, oral treatment with a repurposed PAPD5 inhibitor, the dihydroquinolizinone RG7834, rescued TERC 3' end maturation and telomere length. These findings pave the way for developing systemic telomere therapeutics to counteract stem cell exhaustion in DC, PF, and possibly other aging-related diseases.
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“Small-molecule inhibitors of PAPD5, a non-canonical poly(A) polymerase that destabilizes TERC RNA, have been shown to rescue TERC levels in induced pluripotent stem cells from patients with dyskeratosis congenita190.”
From Telomere dysfunction in ageing and age-related diseases (2022) · cites this paper as [190]
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Cites
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“When primary fibroblasts were transduced with a retrovirus encoding TERT, both BCH001-treated and untreated cells were immortalized, but only cells treated with BCH001 showed telomere lengthening (Figure 4H).”
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Zhan, H., Zheng, R., Li, Y., Liu, X. W., Shi, Y., Chen, R., Hou, Q., Huang, H. F., & Lin, M. (2026) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2026.06.034
Molecular Interplay of PARN and Telomerase: Tail Modifiers and Disease Implications
Felicitus, S., Pinto, S. S., Ramakrishna, S. S. M., Nanjappa, D. P., Chakraborty, G., & Chakraborty, A. (2026) · Wiley Interdisciplinary Reviews - RNA · doi:10.1002/wrna.70049
Telomerase RNA regulates the epigenome primed for human lineage commitmentPreprint
Li, J., Su, P., Gao, M., Liu, C., Li, N., Feng, G., Yu, Y., Chen, Z. Q., Yin, G., Ye, X., et al. (2026) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2026.07.03.736284
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Köseoğlu, G. M., Karaatmaca, B., Metin, A., Bilir, Ö. A., Özbek, N. Y., & Kılıç, E. (2026) · Molecular Syndromology · doi:10.1159/msy/adrag004
Metadata
- Authors
- Nagpal, N., Wang, J., Zeng, J., Lo, E., Moon, D. H., Luk, K., Braun, R. O., Burroughs, L. M., Keel, S. B., Reilly, C., Lindsley, R. C., Wolfe, S. A., Tai, A. K., Cahan, P., Bauer, D. E., Fong, Y. W., & Agarwal, S.
- Affiliations
- Boston Children's Hospital (US)
- Brigham and Women's Hospital (US)
- Broad Institute (US)
- Johns Hopkins University (US)
- University of Massachusetts Chan Medical School (US)
- Seattle Children's Hospital (US)
- Dana-Farber Cancer Institute (US)
- Tufts University (US)
- Corresponding author
- Agarwal, Suneet
- ORCID
- Venue
- Cell Stem Cell
- Publisher
- Elsevier BV
- Published
- 2020-06
- Volume
- 26
- Issue
- 6
- Pages
- 896-909.e8
- ISSN
- 1875-9777 (ISSN-L)
- 1934-5909
- PMC
- PMC7275922
- OpenAlex
- W3017218125
- Cited by
- 105
- References
- 89
- Citations by year
- 2026: 15 · 2025: 15 · 2024: 17 · 2023: 17 · 2022: 22 · 2021: 10 · 2020: 8
- FWCI
- 8.56
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Cell stem cell (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- U.S. Department of Defense
- National Institute of Diabetes and Digestive and Kidney Diseases
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cell Biology › Telomeres, Telomerase, and Senescence
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Aging › Genetics, Aging, and Longevity in Model Organisms
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Nuclear Structure and Function
- Keywords
- telomerase activity, dyskeratosis congenita, pulmonary fibrosis, induced pluripotent stem cells, blood stem cells, xenotransplantation, stem cell exhaustion, age-related diseases
- MeSH terms
- Animals, Humans, Mutation, RNA, Telomere, Telomerase, Dyskeratosis Congenita, Mice, Induced Pluripotent Stem Cells
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