Genetic therapy: on the brink of a new future

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BioMed Central

Open Access

Editorial

Genetic therapy: on the brink of a new future Gary R Hellermann* and Shyam S Mohapatra Address: Joy McCann Culverhouse Airway Disease Research Center, University of South Florida College of Medicine, Div. of Allergy and Immunology, Tampa, Florida Email: Gary R Hellermann* - [email protected]; Shyam S Mohapatra - [email protected] * Corresponding author

Published: 04 September 2003 Genetic Vaccines and Therapy 2003, 1:1

Received: 18 August 2003 Accepted: 04 September 2003

This article is available from: http://www.gvt-journal.com/content/1/1/1 © 2003 Hellermann and Mohapatra; licensee BioMed Central Ltd. This is an Open Access article: verbatim copying and redistribution of this article are permitted in all media for any purpose, provided this notice is preserved along with the article's original URL.

The field of genetic therapeutics, the focus of our new xxx journal, Genetic Vaccines and Therapy, is poised to enter a potentially vast new arena of medical progress within this decade. Worldwide interest exists among biopharmaceutical companies, governments, the military and medical practitioners in the rapid development and deployment of novel DNA-based agents. The possibilities are virtually unlimited: new treatments for emerging and re-emerging diseases; effective and specific immunization against the viral scourges of HIV, smallpox and others; the genetic correction of hereditary defects, such as cystic fibrosis; more effective and specific antitumour therapies genetically tailored to the individual; and gene-specific preventive measures for degenerative diseases such as Alzheimer's, arthritis, and arteriosclerosis [1]. Much good and useful work has been done in the field of genetic medicine, but much remains to be perfected. Gene-based medicine has played a role and will continue to be of critical importance in creating vaccines and antiviral therapies for HIV, hepatitis, herpes and other viruses, in new strategies for the prevention and treatment of emerging diseases, and for a secure and certain biodefense plan against microbial attack. For example, cancer patients may look forward to a swift and specific treatment regimen based on detailed genetic analysis of their tumour type and individual genetic makeup that can be used to design DNA-based antitumour agents with minimal side effects [2]. The burgeoning wealth of new information generated by genome sequencing and gene chip analysis, combined with the enhanced proteomics databases available today opens up huge possibilities for designing new therapeutic

agents. The use of DNA to synthesize proteins with functional or antagonistic properties is unlimited. DNA delivery methods that can potentially target DNA to a specific type of cell or organ, or to a patient's specific tumour type, are under intense investigation. Side effects arising from system-wide application of a drug, as in chemotherapy would be avoided by targeted DNA delivery. Maximum efficacy will be obtained by getting the agent directly to the are