Gene Therapy and Vaccination

William J. Bowers, Michelle C. Janelsins, and Howard J. Federoff 

Keywords Active imimnizadon Adenovims Adeno-associated vkus Alzheimer s disease Amyloid-beta Chemokine Cytokine Lendvirus Herpes simplex vims Isotypes Parkinson s disease Passive immunizadon Single-chain andbodies Thl cells Th2 cells Transgene Vector 

---

Laube BL (2005) The expanding role of aerosols in systemic drug delivery, gene therapy, and vaccination. Respir Care 50(9) 1161-1176. 

M. Schleef, Plasmids for gene therapy and vaccination, Wiley-VCH, Weinheim Germany, 2001. 

As gene therapy and stem cell research progress, we can expect more regulatory requirements to be developed to ensure proper safeguards are implemented. Similarly, xenotransplantation and control of biopharmaceutical products will experience specihe regulatory controls as new advances are made. Exhibit 11.13 presents the FDA s current oversight on gene therapy and its cautious approach to cancer vaccine. 

Clinical trial methodology and guidelines for special disease-related therapeutie fields are discussed. In cooperation with other working parties, guidanee for modified-release oral and transdermal dosage forms, pharmacokinetics, and clinical investigation of new vaccines, gene therapy, and cell-cultured influenza vaeeines has been... 

Every few decades, a medical innovation is perfected that profoundly influences the practice of medicine. Widespread vaccination against common infectious agents and the discovery of antibiotics serve as two such examples. Many scientists now believe that the potential of gene therapy and antisense technology rivals even the most significant medical advances achieved to date. 

Ferreira, G. et al. (2000). Downstream processing of plasmid DNA for gene therapy and DNA vaccine applications. Trendi Biotechnol. 18(9), 380—388. 

The former division now deals mainly with vaccines, gene therapy and blood products. 

Under the revised system, CBER will continue to review blood products and vaccines. These product areas are CBER s strengths and the basis for creation of the biologies division. CBERwill also be responsible for evaluating gene therapy and tissue transplantation products as the development of these novel entities comes to fruition. 

Marconi P, Argnani R, Berto E, Epstein AL, Manservigi R (2008) HSV as a vector in vaccine development and gene therapy. Hum Vaccin 4(2) 91-105... 

Formulation and Delivery in Gene Therapy, DNA Vaccination and Immunotherapy... 

Walsh (2003) defined biopharmaceuticals as therapeutic protein or nucleic acid preparations made by techniques involving recombinant deoxyribonucleic acid (DNA) technology. Therapeutic proteins include blood clotting factors and plasminogen activators, hemopoietic factors, hormones, interferons and interleukins, and monoclonal antibodies (LeVine, 2006). Over time, the term biopharmaceutical has broadened, and, in addition to proteins and nucleic acids, now includes bacteriophages, viral and bacterial vaccines, vectors for gene therapy, and cells for cell therapy (Primrose and Twyman, 2004). Attention here focuses on proteins, since the majority of approved biopharmaceuticals are proteins. 

---