Telomerase gene therapy rescues telomere length, bone marrow aplasia, and survival in mice with aplastic anemia
Open accessAbstract
Key Points: Telomerase gene therapy in a mouse model of aplastic anemia targets the bone marrow and provides increased and stable telomerase expression. Telomerase expression leads to telomere elongation and subsequently to the reversal of aplastic anemia phenotypes.
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At least two mouse models demonstrate a causative role of telomere dysfunction in bone marrow failure.
“At least two mouse models have demonstrated a causative role of telomere dysfunction in bone marrow failure.”
From Telomere dysfunction in ageing and age-related diseases (2022) · cites this paper as [85]
Experimental — accuracy not yet measuredTransplantation of bone marrow from late-generation Tert-knockout mice into irradiated wild-type recipients results in aplastic anaemia.
“Similarly, transplantation of bone marrow from late-generation Tert-knockout mice in irradiated wild-type recipients resulted in aplastic anaemia85.”
From Telomere dysfunction in ageing and age-related diseases (2022) · cites this paper as [85]
In mouse models of bone marrow failure, danazol or AAV-mediated Tert expression improves haematopoiesis.
“In both models, danazol or AAV-mediated Tert expression improved haematopoiesis85,86.”
From Telomere dysfunction in ageing and age-related diseases (2022) · cites this paper as [85]
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- Authors
- Bär, C., Povedano, J. M., Serrano, R., Benitez-Buelga, C., Popkes, M., Formentini, I., Bobadilla, M., Bosch, F., & Blasco, M. A.
- Affiliations
- Telomeres and Telomerase Group, Molecular Oncology Program and
- Human Genetics Group, Spanish National Cancer Research Centre, Madrid, Spain;
- Roche Pharma Research and Early Development, Neuroscience, Ophthalmology, and Rare Disease, Roche Innovation Center and
- Roche Extending the Innovation Network Academia Partnering Program, F. Hoffmann-La Roche, Basel, Switzerland; and
- Centre of Animal Biotechnology and Gene Therapy, Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, Bellaterra, Spain
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2016-04-07
- Volume
- 127
- Issue
- 14
- Pages
- 1770-1779
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2279382765
- Cited by
- 116
- References
- 42
- Citations by year
- 2026: 9 · 2025: 13 · 2024: 23 · 2023: 14 · 2022: 10 · 2021: 8 · 2020: 6 · 2019: 12 · 2018: 11 · 2017: 8 · 2016: 2
- FWCI
- 8.85
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Blood (publishedVersion)
- PubMed (publishedVersion)
- Dipòsit Digital de Documents de la UAB (Universitat Autònoma de Barcelona) (submittedVersion, other-oa)
- Funders
- Ministerio de Economía y Competitividad
- Fundación Botín
- Topics
- Health Sciences › Medicine › Physiology › Telomeres, Telomerase, and Senescence
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Blood disorders and treatments
- Keywords
- aplastic anemia, telomerase reverse transcriptase, telomere shortening, AAV9, hematopoietic stem cells, bone marrow aplasia, pancytopenia, bone marrow hypoplasia
- MeSH terms
- Dependovirus, Anemia, Aplastic, Animals, Disease Models, Animal, Hematopoietic Stem Cells, Transduction, Genetic, Genetic Therapy, Telomere, Mice, Knockout, Telomerase, Mice, Telomere Homeostasis
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