Between 0.6 and 1.8 ng of human factor IX per milliliter was found in mouse plasma, and the production was continued for 23 days in vivo. These results demonstrate a model of ex vivo gene therapy for hemophilia B using gene-modified oral mucosal epithelium....
Therapeutic protein and molecule delivery to target sites by transplanted human stem cells holds great promise for ex vivo gene therapy. Our group has demonstrated the therapeutic benefits of ex vivo gene therapy targeting the skeletal muscles in a trans
Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invent...
Ex-vivo gene therapy of human vascular bypass grafts with E2F decoy: the PREVENT single-centre, r a n d o m i s e d , c o n t r o l l e dt r i a l AD,Donaldson MC,et al.Ex-vivo gene therapy of human vascular bypass grafts with E2F decoy:the PREVENT single-centre, ...
In vivo and ex vivo gene therapy strategies to treat tumors using adenovirus gene transfer vectors. Cancer Chemother Pharmacol 1999; 43 Suppl: S90-S99In vivo and ex vivo gene therapy strategies to treat tumors using adenovirus gene transfer vectors[J] . Ronald G. Crystal.Cancer Chemotherapy and...
Somatic cell-based gene therapy can be widely classified into two categories: • Ex vivo: When gene manipulation is done on cells outside the body, in the external environment and then transplanted back into the patient, such a strategy is called ex vivo gene therapy or gene transfer [114...
Most preclinical studies for hemophilia gene therapy have focused on systemic delivery of viral vectors for in vivo transduction of liver cells [8]. Several such studies have yielded promising results, and long-term therapeutic levels of plasma FVIII in hemophilia A mice have been achieved by syste...
A therapeutic option for monogenic disorders is gene therapy with ex vivo-transduced autologous hematopoietic stem cells (HSCs). Safety or efficacy studies of ex vivo-modified HSCs are conducted in humanized mouse models after ablation of the murine bone marrow and transfer of human CD34+ HSCs. En...
Until recently, progress in ex vivo gene therapy (GT) for human immunodeficiency virus-1 (HIV-1) treatment has been incremental. Long-term HIV-1 remission in a patient who received a heterologous stem cell transplant for acquired immunodeficiency syndrome-related lymphoma from a CCR5(-/-) donor...
Sickle-cell disease (SCD) is caused by an A·T-to-T·A transversion mutation in the β-globin gene (HBB). Here we show that prime editing can correct the SCD allele (HBBS) to wild type (HBBA) at frequencies of 15%–41% in haematopoietic stem and progenit