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Discard Your Genetic Problem Through Gene Therapy
#1
In this decade gene therapy is getting so much attention due to its existing and future probable area of application which is really vast. Till now about 10,000 genetic disorders have been discovered. Among them some of the diseases can be treated through gene therapy right now. Researchers are going on others. Genetic disorders result from abnormalities in genes or chromosomes. Although genetic disorders are quite rare, they can be deeply influential. Some of the disorders which are well known are asthma, autism, autoimmune diseases, cancers, ciliopathies, cleft palate, diabetes, heart diseases, hypertension, inflammatory bowel disease, mental retardation, mood disorder, obesity, refractive error etc.

Gene therapy is a very sophisticated branch of genetic engineering. In a very general sense you can describe it as a technique for correcting defective genes that are responsible for disease development. There are several approaches of it like abnormal gene can be inserted to compensate for a nonfunctional gene or an abnormal gene can be repaired through selective reverse mutation etc. Gene therapies are of mainly two types: germ line cell therapy & somatic cell gene therapy. In the germ line cell therapy process, germ cells (sperm & eggs) are modified by the insertion of functional genes which are integrated into their genome. The result of this therapy is heritable to the next generation. But due to some ethical issues presently this type of therapy is restricted to human. Somatic gene therapy is performed through transferring the therapeutic genes into somatic cells of a patient and the effect of therapy is inheritable to the future generation.

The mechanism of gene therapy actually includes three steps. Firstly a vector delivers the therapeutic gene into a patient’s target cell. Then target cells become infected with the viral vector. And finally the vector’s genetic material is inserted into the target cell. Two different strategies are used for gene delivery i.e. Ex-vivo and In-vivo.

Viruses play an important role in gene therapy as vector. As they replicate by inserting their DNA into host cell gene therapy can use viruses to insert genes that encode for a desired protein to create the desired trait. Mainly Four types of viruses are used in purpose currently. They are retroviruses, adenoviruses, adeno-associated viruses and herpes simplex viruses. Besides this several non-viral options are also available for gene delivery. Amongst them the simplest is the direct introduction of therapeutic DNA into target cells. Another option is the creation of an artificial lipid sphere with an aqueous core. This liposome carrying the therapeutic DNA is capable of passing the DNA through the target cell’s membrane. Third option is to insert therapeutic DNA into target cells by chemically linking the DNA to a molecule which will bind to special cell receptors. Now researchers are trying to introduce 47th chromosome into host cell.

Gene therapy had been started in 1880 on research level. The 1st gene therapy was performed on September 14th, 1990. A patient was treated for Sever Combined Immunodeficiency (SCID). But this work was functional for a few months. But now it is possible to get 90% success. Currently researches are going on a new gene therapy approach which repairs errors in messenger RNA derived from defective genes. If this approach becomes successful then it is possible to treat blood disorder thalassaemia, cystic fibrosis and some cancers for sure. Huntington disease can be treated using RNA interference.

Although human gene therapy products are still not available for sale but its potential and importance is going to make it the exciting and new era of human disease treatment. Hope within a very short time we will see it’s magic. Just wait & watch!
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#2
Germline therapy has many more concerns about it. While gene therapy is only altering the genome of single cells for a time, germline therapy involves permanent alteration of the human gene pool. The introduced gene is carried into all the cells of the child. The concerns previously mentioned are intensified. It is temporary, is perfectly ethical to treat genetic diseases, such as cancer, etc.
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