Gene Therapy: Introduction and Overview

Gene therapy for hemophilia?

“…This confers many more options for trading off ‘idealized’ in vivo protein expression in order to achieve a wider theoretical safety margin. Further, it sustains a vital series of scientific competitions (parallel to that of viral vectors between each other vs. physical means of gene transfer) [9] for best therapeutic strategy in preclinical models. This should enhance the likelihood that therapies worthy of clinical trials really will represent ‘survival of both the fittest and the safest ‘‘strategies’’ for curing hemophilia’.…”