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

22 Feb 2016

Abstract excerpt

Aplastic anemia is a fatal bone marrow disorder characterized by peripheral pancytopenia and marrow hypoplasia. The disease can be hereditary or acquired and develops at any stage of life. A subgroup of the inherited form is caused by replicative impairment of hematopoietic stem and progenitor cells due to very short telomeres as a result of mutations in telomerase and other telomere components. Abnormal telomere...

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Share to RNA Interference and Gene Delivery
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PMID: 26903545
DOI: 10.1182/blood-2015-08-667485

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