Importantly, selective and partial deletion of TRF1 in the BM of TG mice led to rapid telomere shortening, replicative senescence, BM failure, and a poor survival rate (55% at 100 days post viral injection) in the absence of any TERT gene transfer, whereas mice receiving TERT-expressing AAV9 showed a greatly improved survival rate (87%) due to the lower incidence rate of aplastic anemia (empty versus TERT vectors: 44% and 13%, respectively). The gene discussed is TERT; the disease is idiopathic aplastic anemia.