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Diagnostics and bioinformatics

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Biosensors, biomonitoring

Gene transfer is a relatively new possibility for the treatment of rare genetic disorders and common multifactorial diseases. The technique, often called gene therapy, is used to correct defective genes responsible for disease development.

Researchers may use one of several approaches for correcting faulty genes: 1) A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common. 2) An abnormal gene could be swapped for a normal gene through homologous recombination. 3) The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. 4) The regulation (the degrese to which a gene is turned on or off) of a particular gene could be altered.

Typically gene transfer involves using a vector such as a virus to deliver a therapeutic gene to the appropriate target cells. Besides virus-mediated gene-delivery systems, there are several nonviral options for gene delivery such as the direct introduction of therapeutic DNA into target cells; passing the DNA through the target cell's membrane in liposome, which carries the terapeutic DNA, etc. But gene therapy is still in its infancy and is not yet available outside clinical trials. There are some factors, which keep gene therapy from becoming an effective treatment for genetic diseases, e.g. immune response, problems with viral vectors, etc.