Genes therapy technology, advances

The future may hold more advanced technologies that utilize gene therapy as an inhibitor of restenosis. Studies have documented that local gene... 

Every few decades, a medical innovation is perfected that profoundly influences the practice of medicine. Widespread vaccination against common infectious agents and the discovery of antibiotics serve as two such examples. Many scientists now believe that the potential of gene therapy and antisense technology rivals even the most significant medical advances achieved to date. 

L Severe combined immunodeficiency (SCID) syndromes are excellent models for gene therapy because of the genetic basis of these disorders and significant advances in the technology to transfer therapeutic genes into hematopoietic precursor cells. For all these reasons, which of the following syndromes represents an ideal candidate for gene therapy ... 

Cullis, P.R., Chonn, A. (1998). Recent advances in liposome technologies and their applications for systemic gene delivery. Adv. Drug Deliv. Rev., 30(1-3), 73-83. Audouy, S.A., de LeiJ, L.F., Hoekstra, D., Molema, G. (2002). In vivo characteristics of cationic liposomes as delivery vectors for gene therapy. Pharm. Res., 19(11), 1599-1605. 

The challenge of developing successful delivery technologies for gene therapy is a world removed from the simple, sustained-release oral formulations which were the achievements of the first pharmaceutical scientists to specialize in advanced drag delivery technology. The potential, in commercial terms and in terms of human well-being, is too vast to estimate. 

As the technology to deliver genes into ceils becomes safer and more efficient, advances in gene therapy are likely to be seen. 

The use of nucleic acids as therapeutics is an unprecedented concept, and therefore the regulatory guidelines have evolved simultaneously with the technologies, and continue to do so. Because gene therapy using retroviral vectors is the most advanced of the nucleic acid therapies, regulatory issues and concepts are relatively better formulated in this area (Walters, 1991 FDA Report, 1991). 

Considerable advances have occurred in DDS within the past decade. Extended release, controlled release, and once-a-day medications are available for several commonly used drugs. Global vaccine programs are close to becoming a reality with the use of oral, transmucosal, transcutaneous, and needle-less vaccination. Considerable advances have been made in gene therapy and delivery of protein therapeutics. Many improvements in cancer treatment can be attributed to novel drug delivery technologies. 

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