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Formulation and Delivery of Nucleic Acids

  • H. E. J. Hofland
  • L. Huang
Part of the Handbook of Experimental Pharmacology book series (HEP, volume 137)

Abstract

The aim of gene therapy is to correct diseases at their origin by delivery and subsequent expression of exogenous DNA, which encodes for a missing or defective gene product. Two distinct strategies for DNA delivery can be employed. The ex vivo strategy uses the cells of a patient, into which the DNA is introduced in vitro. The genetically modified cells are selected, expanded and, finally, transplanted back into the patient (BERNS et al. 1995). This is a relatively efficient method for gene delivery. Most disorders, however, require direct gene transfer, i.e., delivery of the nucleic acids directly into the affected tissues in vivo. One of the major challenges for the direct gene-transfer approach is to develop a vehicle that is able to protect the nucleic acids from degradation, while delivering the genes of interest to specific tissues and target-cell compartments.

Keywords

Gene Transfer Cationic Lipid Cationic Liposome Direct Gene Transfer Cholesterol Derivative 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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© Springer-Verlag Berlin Heidelberg 1999

Authors and Affiliations

  • H. E. J. Hofland
  • L. Huang

There are no affiliations available

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