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Gene therapy
Posted Date: 16 May 2008 Resource Type: Articles/Knowledge Sharing Category: How things work
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Posted By: Karthika M Member Level: Gold
Rating:  Points: 1
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Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.
Types of gene therapy
Germ line gene therapy
In the case of germ line gene therapy, germ cells, i.e., sperms or eggs, are modified by the introduction of functional genes, which are ordinarily integrated into their genomes. Therefore, the change due to therapy would be heritable and would be passed on to later generations. This approach, theoretically, should be highly effective in counteracting genetic disorders. However, this option is prohibited for application in human beings, at least for the present, for a variety of technical and ethical reasons.
Somatic cell gene therapy
In somatic cell gene therapy, the gene is introduced only in somatic cells, especially of these tissues in which expression of the concerned gene is critical for health. Expression of the introduced gene relieves/ eliminates symptoms of the disorder, but this effect is not heritable as it does not involve the germ line. At present, somatic cell therapy is the only feasible option, and clinical trials addressing a variety of conditions have already begun.
Broad methods
There is a variety of different methods to replace or repair the genes targeted in gene therapy.
A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common.
An abnormal gene could be swapped for a normal gene through homologous recombination.
The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function.
The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.
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Responses
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| Author: Vidya 23 May 2008 | Member Level: Diamond Points : 2 | useful information
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