Gene Therapy

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Gene Therapy




Germ Line Gene Therapy2

Somatic Gene Therapy2

Delivery Systems2

Viral Gene Therapy2

Non-Viral Gene Therapy3


Parkinson's Disease3

Leukemia Treatment4

HIV/AIDS Treatment4


Social Issues4

Ethical Issues5

Personal Viewpoint6


Gene Therapy


Genes are the main units of heredity, functionally as well as physically. Chromosomes carry these genes. These are responsible for making proteins which are making fundamental part of cellular structure. They are mainly responsible for various life functions ( All individuals transmit gene to the future generation. If genes get any disorder, a number of serious diseases affect the normal life functioning. It may cause cystic fibrosis, muscular dystrophy or thalassemia. Moreover if, such mutant genes interact with the environment they might become more severe like cancer or heart diseases (Mammen et al, 2007).

To deal with such disorders, there is an advanced technology named as "Genes Therapy". It was conceived by United States around 1970s. Gene therapy is an effective technology for treating patients having inherited monogenic disorders with recessive inheritance (Giacca, 2010).


Scope of gene therapy is enormously vast. The abnormality of genes causes a number of diseases and disorders in patients. Treating such cases requires genetic approach, and hence gene therapy is the most progressive idea yet.

In Gene therapy, the therapeutic material gets into the target cells, which exerts the intended effects as it gets active. This could be done by different approaches, like

Replacing a defected gene with a normal one.

Repairing the defected gene through reverse mutation, which in turns normalizes the gene function.

By regulating a gene in terms of keeping the gene turned off or on for a time (Giacca, 2010).


Germ Line Gene Therapy

Germ line is the sequence of cells, which develops into eggs and sperms and passes on to the future generations. In this technique, a normal gene enters into the germ line which in turns corrects the individual receiving, but it has defective effects on the off springs (

Somatic Gene Therapy

Somatic gene therapy is for individuals, suffering from a certain disease at any development stage. This means there is no risk of inheritance to future generations. This is done on parts of organs which lower the risk of loss of the entire organ in case of failure (Nicholas.1988).

Delivery Systems

Viral Gene Therapy

For the therapeutic gene to be more effective, a carrier is used with it which is a virus. The virus is altered genetically to carry normal DNA with it like Retroviruses adenoviruses, adeno-associated viruses and herpes simplex viruses. These viruses cause disease in humans by means of encapsulation and delivery of the gene into cells. Retroviruses integrate themselves into the chromosomes while adenoviruses do not integrate into chromosome, but their DNA is introduced into the nucleus of the cell. It is done by directly injecting into the target tissue or exposing the mutant gene with it in a laboratory. It is then reintroduced into the patient after being corrected (Giacca, 2010).

Non-Viral Gene Therapy

In this technique, an artificially developed chromosome is inserted into the DNA. The inserted chromosome must have encoded genetic information, telomeres and centromeres.

The telomeres are the end covers of DNA keeping it from being eaten away, and ...
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