What Is Gene Therapy?

By Petersen Randall / November 27, 2018

Gene therapy is one of those fields of modern medicine that are currently being under the spotlight and high expectation of scientists and patients are focused onto it. The nature gene therapy refers to various methods used to intervene in the genome of a person suffering from some genetic disorder and attempt correction. A wide range of genetic diseases caused by complete missing of a gene, defect structure of a gene or abnormal functioning of a gene, have been incurable so far with only symptomatic therapy available. Gene therapy raises hopes that in relatively near future many groups of patients, even those struggling with disorders causing early death, will be treated with success.

Methods of applying nature gene therapy

Depending on the type of disorder, some genetic diseases are caused by a gene mutation (a small part of the gene is a defect), while others are based on the complete absence of a given gene. Therefore, scientists are developing three possible approaches to gene therapy. The first one refers to gene insertion. The process of multiplication is used to make a sufficient number of copies of a healthy targeted gene, which are then inserted into the body of a patient via various vectors. Defected genes remain inside the body, but inserted genes will produce sufficient amount of healthy and functional proteins. The other gene therapy method refers to gene modification. This method is currently out of our reach due to technological limits, but the basic idea is to conduct direct correction of a defected gene and put it in order. This method would solve all sorts of gene mutations. Gene replacement is the third methods being developed. The therapy would be based on ways to crop-out defect gene and insert healthy, functional copies instead.

Benefits and risks of natural gene therapy

A wide range of potential benefits of nature gene therapy is obvious, although the full capacity and potential of this curing strategy haven’t been explored completely yet. The list of genetic disorders and the spectrum of diseases caused by faulty genes are large and millions of patients suffering from severe conditions would directly benefit from gene therapy. This would prolong their lives, relieve their symptoms and unburden hospitals from regular symptomatic treatments currently provided to these people. On the other hand, there are skeptic people who oppose this treatment stating that it might lead modern medicine to eugenic and enable genetic optimization of people based on non-medical reasons. Simply put, these people are worried about global abuse of this potent treatment which would provide methods for genome interventions which are not medically justified, but ideological agenda.

Other potentials of gene therapy

All previously mentioned methods of applying gene therapy include children or adults and their somatic genes. Aside from this aspect, gene therapy has the potential to modify germ cells and sex genes, causing changes that would be transferred to the next generations. Although this also has medical indications, consequences and side effects are way too blurred and not explored enough at this moment.

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    Petersen Randall

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