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THE PHARMA REVIEW (OCTOBER 2008)

Gene Therapy for Inherited and Acquired Disorders: A Brief Review

Pavan Malhotra, O.P. Gupta, Archana Parihar, Shakti Bala, Geetika Gupta

Abstract: Certain diseases are inherited while some are acquired due to heritable or acquired abnormalities at the level of gene in the genome of individuals. Gene therapy refers to the correction or inactivation of the faulty gene or introduction of a functional gene to make up for genetic deficiency. The functional gene required for the transfer is obtained by recombinant DNA technology. Transfer of gene is carried out either directly or through a vector. Clinical application of gene therapy is being tried both in experimental animals and human beings. More than 300 clinical trials, for gene transfer in patients suffering from various disorders have been taken up. However gene therapy is still at an investigational stage. It is hoped that in the near future, gene therapy will prove to be an effective modality for treating many inherited and acquired disorders.
 
Introduction
Genes are DNA strands. A gene when it expresses, leads to formation of specific mRNA which in turn leads to synthesis of specific proteins and subsequently the specific functions in the body system are performed. Obviously, a faulty gene will lead to absence or reduced formation of a protein and/or of its function as it is likely to express faulty protein and lead to some acquired disorders like immunological disorders including AIDS, cardiovascular, neurological, metabolic or malignancy. While advances made in molecular and cellular biology describe the proteins that are mediating the biological functions or disease processes, the DNA technology provides access to the genes that control these events and it is now possible to even define exactly which DNA base pair is anomalous. Accordingly, different strategies have been developed to rectify these anomalies at gene level.

 

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