Telomerase gene therapy rescues telomere length, bone marrow aplasia, and survival in mice with aplastic anemia-Reference-Cited by-同舟云学术

Telomerase gene therapy rescues telomere length, bone marrow aplasia, and survival in mice with aplastic anemia

Published:2016-04-07 Issue:14 Volume:127 Page:1770-1779
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Bär Christian¹,Povedano Juan Manuel¹,Serrano Rosa¹,Benitez-Buelga Carlos²,Popkes Miriam¹,Formentini Ivan³,Bobadilla Maria⁴,Bosch Fatima⁵,Blasco Maria A.¹

Affiliation:

1. Telomeres and Telomerase Group, Molecular Oncology Program and

2. Human Genetics Group, Spanish National Cancer Research Centre, Madrid, Spain;

3. Roche Pharma Research and Early Development, Neuroscience, Ophthalmology, and Rare Disease, Roche Innovation Center and

4. Roche Extending the Innovation Network Academia Partnering Program, F. Hoffmann-La Roche, Basel, Switzerland; and

5. Centre of Animal Biotechnology and Gene Therapy, Department of Biochemistry and Molecular Biology, School of Veterinary Medicine, Universitat Autònoma de Barcelona, Bellaterra, Spain

Abstract

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.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/127/14/1770/1392759/1770.pdf

Reference43 articles.

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4. Immune mechanism of aplastic anemia.;Nakao;Int J Hematol,1997

5. Inherited bone marrow failure syndromes.;Dokal;Haematologica,2010

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