Abstract

Gene therapy is the attempt to treat diseases by means of genetic manipulation. Numerous challenges remain to be overcome before it becomes available as a safe and effective treatment option. Retroviruses and adenoviruses are among the most commonly used viral vectors in trials. The retrovirus introduces the gene it carries into the target cell genome while the adenovirus introduces the gene into the target cell nucleus without incorporating it into the target cell genome. Other viral vectors such as adeno-associated viruses, pseudotyped viruses and herpes simplex viruses, are also gaining popularity. Proposed non-viral methods for gene transfer include physical methods and the employment of chemical vectors (lipoplexes, polyplexes and inorganic nanoparticles). Recent studies have investigated potential applications of gene therapy in correcting genetic diseases, treating malignant disorders and for treatment of other diseases. Trials on gene therapy for SCID and Leber's congenital amaurosis have achieved considerable success, but the widely publicized adverse reaction in X-linked SCID patient receiving gene therapy raised concerns for safety profile of gene therapy. For that, several methods of improving safety and efficacy of gene therapy have been proposed. At present, the three main gene therapy strategies for treatment of cancer are application to oncolytic viruses, suicide-gene therapy and gene-based immunotherapy. Gendicine, the first approved anticancer drugs based on the use of gene therapy principle, is based on the use of oncolytic viruses. More evidence for wider clinical applications of gene therapy are expected as more gene therapy studies progress from the preclinical phase to clinical trial.

Original languageEnglish
Pages (from-to)188-194
Number of pages7
JournalWest Indian Medical Journal
Volume60
Issue number2
Publication statusPublished - Mar 1 2012

Fingerprint

Genetic Therapy
Oncolytic Viruses
Inborn Genetic Diseases
Retroviridae
Adenoviridae
Genes
X-Linked Combined Immunodeficiency Diseases
Leber Congenital Amaurosis
Genome
Safety
Dependovirus
Simplexvirus
Cell Nucleus
Immunotherapy
Nanoparticles
Suicide
Therapeutics
Clinical Trials
Viruses

Keywords

  • Adenovirus
  • Gene therapy
  • Nonviral vectors
  • Oncolytic viruses
  • Retrovirus
  • Suicide gene therapy

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Tani, J., Faustine, & Sufian, J. T. (2012). Updates on current advances in gene therapy. West Indian Medical Journal, 60(2), 188-194.

Updates on current advances in gene therapy. / Tani, Jowy; Faustine; Sufian, Jomiany Tani.

In: West Indian Medical Journal, Vol. 60, No. 2, 01.03.2012, p. 188-194.

Research output: Contribution to journalArticle

Tani, J, Faustine & Sufian, JT 2012, 'Updates on current advances in gene therapy', West Indian Medical Journal, vol. 60, no. 2, pp. 188-194.
Tani J, Faustine, Sufian JT. Updates on current advances in gene therapy. West Indian Medical Journal. 2012 Mar 1;60(2):188-194.
Tani, Jowy ; Faustine ; Sufian, Jomiany Tani. / Updates on current advances in gene therapy. In: West Indian Medical Journal. 2012 ; Vol. 60, No. 2. pp. 188-194.
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