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Gene Therapy to Fight Genetic Diseases

written by: Rishi Prakash•edited by: Leigh A. Zaykoski•updated: 11/12/2008

This article discusses genetic diseases and the applications used to treat them.

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    Introduction

    Every human being carries defective genes; however, these genes can remain unproductive for several years. In some people, though, these genes might express and cause genetic diseases. With the rapid progression in the field of science and technology, scientists and researchers are developing molecular and genetic methods to fight such problems.

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    Gene Therapy

    One of the most advanced genetic applications in medicine is gene therapy. It involves the insertion of genes in diseased patients so as to treat a disease by replacing a defective mutant allele with a functional one. Moreover, gene therapy provides the most up-to-date research in the field of genetic and cell-based technologies for the treatment of various diseases. Some of these genetic diseases are Huntington's chorea, Duchenne muscular dystrophy, and hemophilia, among others.

    In metabolic genetic diseases, defective genes stay ineffective in catalyzing a particular metabolic reaction. In order to make them functional, gene therapy is used, and the defective genes are removed by working genes. Before treatment, though, a precise diagnosis of the genetic defects needs to be ascertained.

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    Pinpointing Diseases

    Genetic engineering has come up with a powerful tool that can pinpoint specific diseases quickly and accurately. Gene therapy is the most advanced tool being used to cure several genetic diseases. It can be broadly classified into two categories: ex vivo and in vivo.

    Ex vivo is external, where cells are customized outside the body and then transplanted back in again. In vivo is internal, where genes are altered in cells within the body.

    Ex vivo: In this technique, cells from the affected tissue area are removed surgically, new DNA is injected into the cells to correct the disease, and they are allowed to grow in cultures. When the cultures are fully grown, the cells are placed back into the affected area of the patient.

    In vivo: This technique does not require any surgery or anesthesia. In this case, the therapeutic DNA is inserted into the body cells using virus vectors called retroviruses. These are infectious particles that contain the RNA genome packaged in a protein capsid and surrounded by a lipid envelope). Retroviruses are safe and effective in replacing the diseased gene.

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    Other Techniques

    Other genetic techniques are also available for fighting genetic diseases, but these techniques are not frequently used. These methods include Gene splicing (a method in which the DNA of a gene is cut, using restriction enzymes, and the infected portion is repaired). Splicing is used to help produce insulin and other hormones effectively cure human maladies. Gene silencing on the other hand is a genetic therapy method which is also called antisense technology. As the name suggests, this technique inactivates the defective gene. This method is used to treat viruses such as AIDS, Herpes, Hepatitis, and Chicken Pox.

    Scientists are working in the field of Bioinformatics, an applied science that will facilitate the diagnosis of defective genes from the entire human genome.