By David V. Schaffer, Weichang Zhou
1 D.V. Schaffer, W. Zhou: Gene treatment and Gene supply structures as destiny Human Therapeutics.- 2 J. Heidel, S. Mishra, M.E. Davis: Molecular Conjugates.- three M. Manthorpe, P. Hobart, G. Hermanson, M. Ferrari, A. Geall, B. Goff, A. Rolland: Plasmid Vaccines and Therapeutics: From layout to Applications.- four S.R. Little, R. Langer: Non-Viral supply of melanoma Genetic Vaccines.- five J.C. Grieger, R.J. Samulski: Adeno-Associated Virus as a Gene remedy Vector: Vector improvement, construction and scientific Applications.- 6 J.H. Yu, D.V. Schaffer: complicated concentrating on suggestions for Murine Retroviral and Adeno-Associated Viral Vectors.- 7 N. Loewen, E.M. Poeschla: Lentiviral Vectors.- eight N.E. Altaras, J.G. Aunins, R.K. Evans, A. Kamen, J.O. Konz, J.J. Wolf: creation and formula of Adenovirus Vectors.-
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Extra resources for Gene Therapy and Gene Delivery Systems
Sample text
4 Technology reviews . . . . . . . Plasmid design . . . . . . . . Structural elements . . . . . . . Components for eukaryotic cell expression Future plasmid design for human use . . Plasmid manufacturing . . . . . . Plasmid delivery systems and formulation Plasmid administration: Devices . . . . . . . . . . . . . . . . . . . . . . . . . . . 9 Case study: From concept to clinic . . . . . . . . . Anthrax vaccine case study . . . .
Pre-IND meeting and preclinical studies and IND application . Conclusion of the case study . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 75 77 77 78 80 81 81 81 83 83 5 Conclusions . . . . . . . . . . . . . . . . . . 83 References . . . . . . . . . . . . . . . . . . . . 84 . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
This is a difficult problem to address in preclinical and clinical studies since the structure(s) of plasmid in the nucleus following cell entry is unclear and the best structure of the plasmid (covalently closed circle, nicked circle, or linear) for effecting the highest level of transcription is also unknown. The ability of the transcribed mRNA to be transported from the nucleus to the cytoplasmic ribosome is known to be affected by the presence of an intron on the primary transcript and the strength of processing (intron removal).
