Gene therapy

Mission

At Firstcure Cancer and Medical Centre, we strive to deliver powerful and innovative therapies to improve the lives of patients and their families.

Introduction to Gene Therapy

Gene Therapy refers to the introduction of genetic material into target cells of a patient to modify and control protein expression for either therapeutic or experimental purpose. A gene therapy medicinal product is a biological product whose features are a) contains an active substance that comprises a recombinant nucleic acid administered to patients with the aim of repairing, regulating, adding or eliminating a genetic sequence; and b) its therapeutic, prophylactic or diagnostic effect associates directly to the recombinant nucleic acid sequence it has or to the product of genetic expression of this sequence.

Gene therapy treatment entails of the insertion, removal or alteration of a therapeutic or working gene copy to cure an illness or defect, or to slow the progression of an illness.

Gene therapy is used for the treatment of many diseases such as cancer diseases, neurological diseases, monogenic diseases, cardiovascular diseases, inflammatory diseases, infectious diseases and ocular diseases. The diseases include those inherited through a genetic disorder (that includes hemophilia, human severe combined immunodeficiency, hereditary blindness, cystic fibrosis, Adenosine deaminase (ADA) deficiency, sickle cell disease, etc.) or acquired (including Parkinson's disease, AIDS or cancer). Below is diagram of indications treated by gene therapy clinical trials.

Indications addressed by gene therapy clinical trials (Source Villate-Beitia, et al., 2015).

Effectiveness of Gene therapy

The success of gene therapy process is attributed to proper transfection vectors that should be able to safeguard nucleic acids against degradation by blood and interstitial nucleases, release the nucleic acids once inside the cell to the proper site and promote internalization of the genetic material into target cells. The effectiveness of gene therapy get governed by the ability of the vector to reach the relevant tissue and when it gets there, to promote the expression of suitable quantities of the gene product. As shown in the graph below numerous clinical trials for gene therapy have been approved worldwide since the onset of the first gene therapy.

Number of gene therapy clinical trials since 1989 (Source Gascón, Del Pozo-Rodríguez & Solinís, 2013)

Benefits and Successes of Gene Therapy

Gene therapy treatment has been determined to be a safe treatment technique for patients of all ages including those as young as eight years. One of the earliest successes in gene therapy arose from gene-marking studies to show the feasibility of introducing DNA sequences into human cells in patients. Treatments of ocular diseases and inherited immune deficiencies have been done successfully using human gene therapy (Herzog, Cao & Srivastava, 2010). The successful treatment of cancer using gene therapy has been achieved by a) replacing mutated tumor suppressor genes, b) delivering the genetic component of targeted prodrug therapies, c) inactivating overexpressed oncogenes, and d) modifying the antitumor immune response.

References

Villate-Beitia, I., Puras, G., Zarate, J., Agirre, M., Ojeda, E., & Pedraz, J. L. (2015). First insights into non-invasive administration routes for non-viral gene therapy. In Gene Therapy-Principles and Challenges. InTech.

Gascón, A. R., del Pozo-Rodríguez, A., & Solinís, M. Á. (2013). Non-viral delivery systems in gene therapy. In Gene Therapy-Tools and Potential Applications. InTech.

Herzog, R. W., Cao, O., & Srivastava, A. (2010). Two decades of clinical gene therapy–success is finally mounting. Discovery medicine, 9(45), 105.

Carolyn Morgan is the author of this paper. A senior editor at MeldaResearch.Com in Online Paper Writing Service. If you need a similar paper you can place your order from Professional Custom Writing Services.