BACKGROUND Chimeric antigen receptor (CAR) T cell immunotherapy has resulted in
complete remission (CR) and durable response in highly refractory patients. However …

Integrating vectors are associated with alterations in cellular function related to disruption of
normal gene function. This has been associated with clonal expansion of cells and, in some …

Abstract Hematopoietic Stem Cell (HSC) gene therapy (GT) may provide lifelong
reconstitution of the hematopoietic system with gene-corrected cells1. However, the effects …

Liver-directed gene therapy for the coagulation disorder hemophilia showed safe and
effective results in clinical trials using adeno-associated viral vectors to replace a functional …

Gene therapy (GT) has rapidly attracted renewed interest as a treatment for otherwise
incurable diseases, with several GT products already on the market and many more entering …

Mobilized peripheral blood is increasingly used instead of bone marrow as a source of
autologous hematopoietic stem/progenitor cells for ex vivo gene therapy. Here, we present …

Quiescent human hematopoietic stem cells (HSC) are ideal targets for gene therapy
applications due to their preserved stemness and repopulation capacities; however, they …

Hematopoietic stem cell gene therapy (GT) using a γ-retroviral vector (γ-RV) is an effective
treatment for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency …

Hemophilia A (HA) is a rare bleeding disorder caused by deficiency/dysfunction of the FVIII
protein. As current therapies based on frequent FVIII infusions are not a definitive cure, long …

Viral vectors are used to insert genetic material into semirandom genomic positions of
hematopoietic stem cells which, after reinfusion into patients, regenerate the entire …