Abdominal Key

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,…

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…

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…

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…

Disease Reference Nonalcoholic steatohepatitis [21] Hepatitis [22, 23] Fulminant hepatitis [11, 24] Liver injury [11, 25] Liver fibrosis [11] Rejection after the transplantation [26] Obesity [11, 27, 28] Diabetes [11,…

Fig. 3.1 Proposed mechanism of liver regeneration by bone marrow cell (BMC) infusion Fig. 3.2 Overview of “study on the efficacy and safety of autologous bone marrow cell infusion therapy…