Gene Therapy Strategies in general

In this post, we are going to talk about several types of gene therapy strategies. One of the gene therapy strategies would be Gene Augmentation Therapy (GAT). GAT introduces extra duplicates of the normal gene to cure diseases that are affected by the dysfunctional gene. The amount of normal gene will rise to a level where the protein is restored in the body cell (Tankonyvtar.hu, 2016). For instance, by creating a functional duplicate of the DNA to fix the disease, gene augmentation therapy can be used to cure cystic fibrosis (Yourgenome.org, 2016). The illustration below explains how GAT works.

gene_therapy_augmentation_yourgenome

Photo credits: www.yourgenome.org

Besides, another type of gene therapy strategy is targeted killing of specific cells. This strategy is to treat certain groups of cells which are destroyed, by inserting DNA into a diseased cell which will cause the cell to die. For instance, treating cancer commonly (Yourgenome.org, 2016).

Furthermore, there are direct and indirect ways to achieve killing of specific cells. Direct cell killing generates a lethal toxin (suicide genes) into the gene to kill the conferring weakness by a subsequently controlled drug. On the other hand, indirect cell killing produces and improves an unaffected response against the affected cell by using immune-stimulatory genes (Yourgenome.org, 2016).

gene_therapy-03

Photo credits: http://www.yourgenome.org

Last but not least, targeted inhibition of gene expression is also one of the gene therapy strategies. It uses different types of system to stop the expression of a particular gene at the DNA, RNA or protein levels when the disease cells show an unusual gene product (Tankonyvtar.hu, 2016). For example, they used gene inhibition therapy to treat cancer by removing the movement of the oncogene. Hence, it can prevent the cell keep growing and stop the disease (Yourgenome.org, 2016).

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Photo credits: http://www.yourgenome.org

Written By: Ke Hong Yee

References

Tankonyvtar.hu. (2016). Molecular therapies. Digital Textbook Library. [online] Available at:http://www.tankonyvtar.hu/en/tartalom/tamop425/0011_1A_Molekularis_terapiak_en_book/ch03s03.html [Accessed 30 Sep. 2016].

Yourgenome.org. (2016). What is gene therapy?. [online] Available at: http://www.yourgenome.org/facts/what-is-gene-therapy [Accessed 30 Sep. 2016].