Biomedical Engineering Reference
In-Depth Information
3
Gene Delivery Using Physical
Methods
Kaustubh A. Jinturkar, Mohan N. Rathi,
Ambikanandan Misra
Pharmacy Department, TIFAC - Centre of Relevance and Excellence in New
Drug Delivery Systems, The Maharaja Sayajirao University of Baroda,
PO Box 51, Kalabhavan, Vadodara 390 001, Gujarat, India
3.1 Introduction
The study of gene transfer to bacterial and mammalian cells has been a major
research area of modern biotechnology and molecular biology. The last two decades
have witnessed rapid development in human genome research and delivery technolo-
gies, with successful gene delivery in vitro , in vivo , and in clinical trials. The earlier
research experiments successfully demonstrated uptake and expression of exogenous
DNA into mammalian cells, and scientists have applied this approach of gene ther-
apy research to the treatment of many human diseases. The classical concept of gene
therapy, delivering the functional gene to correct the missing or mutated gene and to
cure genetic disorders (e.g., adenosine deaminase deficiency and Duchenne muscu-
lar dystrophy) and acquired disorders (e.g., cancer), by promoting the expression of
normal protein and restoring the correct cellular function is already being tested in
humans, although there are still major obstacles to overcome [1] . Ideally, gene deliv-
ery methods should have specificity for the target diseased cell, should protect the
transgene against metabolic degradation by nucleases in intercellular matrices and
against immune attack, should transfer the transgene across the plasma membrane
and into the nucleus of target cells for appropriately regulated transgene expression
and desired safety profile with minimum side effects [1,2] . With innovations in DNA
delivery technology and molecular biology, the gene therapy for treatment of genetic
and hereditary diseases and the DNA vaccination have promoted gene delivery as an
integral strategy along with other treatments in designing and development of effec-
tive therapeutic treatment of hereditary human diseases. But still, the biggest chal-
lenge of gene therapy is to develop a method for successfully delivering the desired
exogenous therapeutic gene (transgene) to the selected defective cells for proper gene
expression at desired rate and extent. Hence, it is essential to develop the safe gene
therapy methods with sufficient targeting ability and high transfection efficiency,
before the gene therapy could be used in clinical practice.
In general terms, gene delivery methods for optimal delivery and expression of
exogenous DNA to the desired mammalian cells in vitro and in vivo are subdivided
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