Results from the single-arm open-label trial have been able to elicit an extension of CD34+ cells ex vivo in two out of the 12 patients enrolled on the trial. The trial data, published in the New ...

In the study, "Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A," published in The New England Journal of Medicine, researchers used a lentiviral vector to deliver gene ...

“Gene therapy for hemophilia A with the use of lentiviral vector-transduced autologous HSCs resulted in stable factor VIII ...

Pain and clinical scores were calculated for each patient. Image analysis to calculate area percent for TLR-2 and CD34 immuno-expression was performed. Data was tabulated and statistically analyzed.

With gene therapy, CD34+ cells can produce fetal haemoglobin, which then takes over from the faulty version. Yen carries out in vivo pharmacology and toxicology studies on these therapies in mice ...