A different type of somatic gene therapy has been introduced for the treatment of diseases that are not primarily caused by inherited genes, such as AIDS and cancer. Scientists were successful in this technique after hundreds of clinical trials. A defect in a somatic cell only affects a person, but if a defected gene is found in a germ cell, there are hundred per cent chances that the defective gene is granted transfer into the next generation. In somatic gene therapy, the desired gene is inserted inside the somatic cell, which is later expressed into therapeutic gene products, it has a great impact on a patient to cure both inherent and acquired diseases.
Isolation of healthy and desired gene from the disease-free person.
Extract somatic cells (having defected gene) from the patient body, culture it.
After isolation, the DNA fragment is subjected to restriction endonuclease enzyme, this enzyme cuts the DNA fragment, and the desired DNA fragment or gene is now separated from the DNA fragment.
Desired gene or DNA fragment is not directly inserted into the patient cell because it does not become functional.
To deliver a gene, a carrier molecule is required, called a vector. A vector may be a plasmid DNA or virus; in that case, animal viruses are often used as a vector because they can hack the machinery of cells by infection. But certain viruses are used for this purpose like retroviruses and adenoviruses. Remember one thing before inserting a virus as a vector into living cells, scientists remove its virulence elements through non-viral transfection method. Liposome and biolistic methods can be used in both in-vivo and ex-vivo gene therapy.
The vector can be inserted into specific tissues or cells, from where it is taken up by individual cells.
Then, cultured cells are transfused into the patientâ€™s body.
Inside the cells, the desired gene is expressed into a therapeutic product.
After a couple of times, patients start to recover from the genetic disease.
Somatic gene therapy may be classical or non-classical gene therapy. In classical gene therapy, specific genes are inserted into the target cells to obtain optimal expression of inserted genes, and the idea of non-classical gene therapy is a process to prevent the expression of genes linked with pathogenesis, to remove the genetic defect and restore the normal gene expression.
Success story of somatic gene therapy
The first human to receive gene therapy was a four-year-old girl, who was suffering from severe immune deficiency disease (adenosine deaminase enzyme deficiency). This defect is caused by a defected or inactive gene that is unable to express or encode proteins or enzymes.
Firstly, some white blood cells (WBCs are somatic cells) were extracted from the little girl. Then these cells were exposed to a genetically engineered virus with a healthy gene. The virus was inserted into the white blood cells, then these cells were transfused back into the girl. Once processed, the white blood cells rebuilt in the bloodstream, the cells started producing therapeutic enzyme. After this treatment, the girl needed regular insertion of white blood cells with the desired gene, because this case gene therapy was applied to somatic cells.
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