[ExpertScie]

As the name suggests “Gene Therapy”, uses gene or genetic material (DNA) for treating specified disease. Gene therapy is nothing but introducing functioning gene to the cell or tissue and thus the effect of dysfunction of cell can eliminated and it restore to normal with the help of this functioning gene. Gene therapy are now a day’s used to treat many disease including combined immunodeficiency syndromes, Hemophilia, many types of cancers, Muscular Dystrophy, Sickle cell anemia etc..
Gene therapy is of two main types and can be categorized as Germ line cell gene therapy which results in a permanent change and can pass down to the next generation also. It is possible by using Germ line therapy to eliminate many hereditary diseases from the families, but it ethical issues needs to be considered while doing so. Second is the Somatic cells gene therapy, which is restricted to individual and is non permanent. These treatments do not pass to next generation. This is a repetitive therapy in which an individual needs to take somatic functional cells again and again by various means.
Basic issue with the gene therapy is nothing but to introduce the functioning gene to the targeted area, Various methods are being used do this and includes but not limited to use of vectors or viral (transduction), non-viral vectors (transfect ion) etc.
Common methods used in gene therapy are - Gene augmentation therapy (GAT), Targeted killing of specific cells, Targeted mutation, Targeted expression inhibition of gene. Further with the advancement in biotechnology, genetic engineering and cell-biology new methods are now used in gene therapy to treat diseases.
Recently persons own cell are used to treat genetic disorders, one such is case of Friedreich’s ataxia which uses Tal effectors (TALE) proteins which helps in targeting specific DNA and thus helps in reducing the effects of disease. Nano technology is also being used in this therapy especially by creating Nano-particles of various shapes. These are prepared from protective polymer coating and used as a non-viral vector which will deliver DNA in targeted cells and will help to eliminate the diseases specially cancer. Using Gene therapy even obesity can be treated successfully.
This type of advancement in gene therapy is really helping us to eliminate many diseases from our life!