How does gene therapy work?
Approaches to gene therapy:
- Insertion - a normal gene may be inserted into the genome to replace a nonfunctional gene; the most common method of gene therapy
- Reverse mutation - an abnormal gene can be repaired by mutating the gene to return it to normal
- Regulation - DNA can be manipulated to causes a gene to turn on or off to certain degrees
Picture Source:
http://www.genengnews.com/insight-and-intelligenceand153/
clinical-successes-and-new-technologies-revive-gene-therapy
/77899451/
A method of gene insertion: vectors
- To insert a new gene to replace a dysfunctional gene requires a carrier, called a vector, to transport and install the normal gene into the abnormal cells.
- Most commonly viruses are used for this job, because they are pathogenic. They insert their DNA into human cells and often cause disease.
- The viruses are changed to carry human DNA, and the new genetic code is able to take over the cell. For gene therapy purposes, viruses deliver healthy DNA to the cells and manipulate the genome to return to normal.
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/thumb/3/3d/Gene_therapy.jpg/300px-Gene_therapy.jpg
Why is gene therapy significant?
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| Picture source: http://www.geneticmutation.net/ |
- Gene therapy is significant because it has the power to change genetic code.
- This can be used to reverse illnesses or to change the physical characteristics or internal processes of a person.
- Gene therapy could be especially useful is in the area of genetic disorders.
- Genetic disorders are caused when genes are mutated, or changed.
- Some mutations are beneficial, but others alter essential body processes.
- There are four types of genetic disorders, and the most common are single-gene disorders, which have mutations in one gene of the DNA sequence. There are over 6,000 genetic disorders of this type that are known.
If we can develop gene therapy more, it is possible that we can improve the lives of millions who suffer under these genetic diseases.


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