Gene therapy can treat genetic disorders by replacing a mutated gene that causes disease with a healthy copy of the gene. Viruses are often used as vehicles to deliver replacement genes into cells, but they can cause immune reactions. Liposomes are now seen as a better alternative as they can carry genes into cells without immune responses. Gene therapy aims to cure monogenic recessive disorders by adding a missing healthy gene, while dominant disorders may be treated through RNA interference to silence mutated genes.

1. Gene therapy summary

2. There is a number of diseases that can’t be treated with specific drugs or antibiotics
because of their genetic nature. Genetic disorders involve a wide range of disasters
caused by the improper functioning of a single gene or several genes. Drugs and
antibiotics used by the classical medicine can just facilitate the state of the patient, they
can’t cure him. The only field of medicine that can deal with genetic disorders when they
have already developed in a person is gene therapy (Touchefeu Y. et al., 2010).
Gene therapy is usually used to deal with monogenic disorders, like cystic fibrosis,
polydactyly, sickle cell anaemia, thalassaemia and others. In the classical approach
gene therapy involves the isolation of a normal target gene, its insertion into a virus
vector with reduced pathogenic abilities and delivery of the gene into target host cells by
the virus. In the case of cystic fibrosis, for example, an adenovirus delivery system is
used (Xiao P.J. et al., 2012). Besides adenoviruses, retroviruses, adeno -associated
viruses and herpes simplex viruses can be used for gene delivery. But there are some
ethical and moral issues coming from using viruses as delivery systems. That’s why
new methods to bring the DNA into the cell were developed. Liposomes are small lipid
granules carrying the target genes. They don’t cause immune reactions and considered
to be a better delivery system than a virus (Patil P.M. et al., 2012).

3. The ways described are used when the disease is a recessive disorder. But if it is a
dominant disorder, gene therapy uses methods of RNA silencing to inactivate the genes
involved into the disease development. Specific synthetic RNA molecules can be
delivered into the cell, via the methods discussed, to disrupt the expression of the
disrupted gene. Besides of monogenic disorders curing gene therapy has been…
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