Implantation of autologous skin fibroblast genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients.

摘要: OBJECTIVE To examine the safety and effects of gene therapy for hemophilia B by implantation autologous fibroblasts genetically modified to secrete clotting factor IX (hFIX). PATIENTS AND METHODS Two patients LD LW were selected from one family accept transfer study. The hFIX protein both about 100 ng/ml plasma activity was 2%. skin two retrovirus-mediated with XL-IX N2CMVIX vector (HBSF-IX). Human measured ELISA, one-stage assay barium citrate sorbent method. inhibitor assayed Bethesda methods. cDNA detected PCR. HBSF-IX cells mixed collagen injection after assessments. RESULTS secreted at high levels in vitro. After cells, no treatment-related side observed. Plasma increased over 2 folds several injections persisted more than 420 days. Blood significantly patients, hemorrhagic tendencies have been partially corrected treatment. Further elevation can be achieved repeating same treatment days later Patient LD. CONCLUSIONS Implantation fibroblast human offers a simple, safe effective approach B.