Fig. 13.1 Investigational New Drug (IND) vial-ampoule manufacturing machine Clinical Scale vialampoule aseptic processing isolator system 13.2.1 Quality Control of Sterile Drugs When validation was first introduced in the 1970s,…

Fig. 4.1 The kinetics of TRAIL expression on liver NK cells after hepatectomy. (a) The numbers presented are means ± SEM of the TRAIL-positive cell population in NK cell subsets. *p < 0.01 for…

Disease Trial site Published year Reference ADA-SCID gene therapy NCI, Hokkaido U 1990 [1] X-linked chronic granulomatous disease (CGD) NIH, Frankfurt, Seoul 1977 [2] Gene therapy of X-SCID disease INSERM…

Autologous Allogeneic Donor disease issues may require alternatives Prospective donor selection can reduce genetic risk Expensive to manufacture Only iPSC-based source Much cheaper but cost of immune suppression Much longer…

Fig. 7.1 Two types of gene loading to HAC. The gene of interest, isolated in a circular vector, is introduced into the HAC by site-specific insertion (above). A megabase-size gene…

Advantages Disadvantages Safety based on non-pathogenicity Limitation of transgene cassette length (up to 5 kb) Wide range of target tissue Long-term transgene expression High prevalence of neutralizing antibody Stability of…