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What is gene therapy
#1
Gene therapy is the insertion of healthy genes into the body of the person in place of diseased genes to cure the disease. Mutations, hereditary diseases and some of the acquired diseases like cancer can be treated with gene therapy. Hemophilia is one of the hereditary diseases on which scientists are doing research so that it can be treated with gene therapy. Though gene therapy has not yet gained recognition but there are some successful cases treated with gene therapy on small scale.
Genes are the basic units of the human body which carry all the traits and characteristics of the person. They are located on chromosomes and are encoded in a special way to make proteins. When some type of mutation occurs in genes due to some reason, then they become unable to perform their normal function and as a result genetic disorders occur. There are many ways through which scientists insert genes in the body to replace faulty genes. For example, a scientist can insert a healthy gene in the body without knowing its location which has the ability to find its place and replace the diseased gene. It is the most common way of inserting the gene. Homologous recombination can be used to replace the abnormal gene with functional gene. Another way of making the gene functional is reverse transcription in which gene can be repaired and perform its normal function.

Process of gene therapy:-
Gene therapy works in following ways.

1) A gene of interest is taken which replaces the diseased gene. To perform this function, a carrier molecule called vector or plasmid is used usually virus is used for this purpose.

2) Vectors have the ability to carry the functional genes to the target cells. Viruses are the microbes which cause infection in the human body. Scientists have availed this opportunity and suppressed its pathogenic activity. This way virus can easily insert the desired gene in place of its pathogenic activity.

3) Some of the target cells like liver or lung cells are treated with viral vectors. When vector reaches the target cells, it releases its genetic material along with the foreign gene and replaces the abnormal cells.

Vectors used in Gene therapy:-
Retroviruses:-
Retrovirus can be used in gene therapy because it has the ability to make double stranded DNA from its RNA genome. This DNA can be incorporated into the host cell.

Adenoviruses:-
This virus contains double stranded DNA and causes respiratory infections. It can also be used as vector.
Similarly herpes simplex virus and adeno associated viruses can also be used as vectors to take the desired genes to the target cells.

Ethical issues in gene therapy:-
Gene therapy if applied on stem cells, it cannot pass to the next generations but if germ line cells are treated with gene therapy, the changes may pass to the next generations. For example, a person having some genetic disorder will not be able to pass its defect to the next generations. But still it is difficult because it may affect the fetus and the side effects may also cause damage to the genes of next generations. Some critics say that gene therapy means that scientists are playing with God because humans have no right to change what God has put in them.
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#2
Gene therapy is latest method in research involving genetic and cell-based technologies to treat diseases.
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