Gene Therapy: A Veracity or Myth.

Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person’s gene. It involves the transfer of a therapeutic or working gene copy into specific cells of an individual in order to repair a faulty gene copy. Thus it may be used to replace a faulty gene, or to introduce a new gene whose function is to cure or to favorably modify the clinical course of a condition. The scope of this new approach to the treatment of a condition is broad, with potential in the treatment of many genetic conditions. Though single gene disorders are best treated than multifactorial disorder; the challenge of developing successful gene therapy for any specific condition is considerable. The problem of ‘gene delivery’ into the desired tissues is very complex and challenging. Some of the ‘vectors’ for delivering the working copy of the gene to the target cells include using harmless viruses and non viral vectors. Till date, in gene therapy, only somatic cells and not the germ cells are targeted for treatment. The possible genetic manipulation of the germ cells remains the subject of intense ethical and philosophical discussion. Though some devastation was recorded in gene therapy trial; the potential benefits of new treatments must always be balanced against such risks. In particular, safety will appropriately remain an important consideration as the field of gene therapy evolves. The purpose of this review is to focus on merit and demerit of gene therapy and to provide information about its future prospective.

Brugada Syndrome: A Major Cause of Sudden Cardiac Death.

In 1992 a new syndrome was described consisting of syncopal episodes or sudden death in patients with a structurally normal heart and an electrocardiogram characteristic of right bundle branch block with ST segment elevation in leads V1 to V3. Brugada syndrome is an autosomal dominant disorder. It has been shown to be associated with mutations in the gene (SCN5A) that encodes for the sodium ion channel in cardiac myocyte. Over 160 mutations of gene SCN5A have been identifi ed. Th e incidence of the disease is diffi cult to estimate, but it causes sudden deaths of 5 per 10,000 inhabitants per year and involved much more frequently in people of Asian ancestry. Diagnosis can be easily made by means of genetic analysis and ECG. Recent data suggest that loss of the action potential dome in the right ventricular epicardium underlies ST segment elevation seen in this syndrome. Right ventricular epicardium is preferentially aff ected because of the predominance of transient outward current in this tissue. Antiarrhythmic drugs like amiodarone and beta-blockers do not prevent death in symptomatic or asymptomatic individuals. Th ough Implantation of an automatic cardioverter–defi brillator is the only recently proven eff ective therapy; Quinidine has been found to decrease Ventricular fi brillation and could prove to be a secured option of implantable cardioverter–defi brillator. However, researcher set focus on gene therapy that may off er an enduring cure in future years. Th e purpose of this brief review is to record the past highlights that have brought us to our present understanding of Brugada syndrome.