Gene therapy is the process through which healthy genetic code is included in cells to replace abnormal genes or create a desirable protein. Researchers explore various gene therapy strategies to resolve problems such as mutated gene replacement with a healthy gene code. This deactivates any mutated genes and adds a healthy gene into the body to help tackle a genetic disease.
There are two possible mechanisms that could be used to undertake gene therapy: Somatic and germline gene therapy. The scientific method selected depends on the type of cell being treated. Somatic gene therapy requires the transfer of a healthy gene into body cells which are mutated and this mechanism only affects the patient alone. Not, their offspring; somatic cells are not inherited. Therefore this mechanism will only affect the patient on an individual- rather and a species- level. On the other hand, Germline gene therapy technique is similar to somatic therapy although this requires the alteration of the a gametes-generating section of the DNA which is defective. Germline therapy has an impact on the offspring of patients as the gametes would possess a healthy gene which is passed onto the offspring at birth. Hence this technique will have an affect on a species level rather than an individual level this may result in the introduction of new alleles as the patient is able to alter their offspring’s characteristics. This can have a determinatal effect on hemospaiens as selection pressures may select aganist the characteristics being favoured by the parents and the offspring is unable to adapt to the environment. If we change the gamete of an individual its ability to respond to environmental change or pressure may decrease its ability to survive and reproduce.
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Biologist must first identity the genetic defect that occured in the gene sequence which caused the disease to occur. We must use a vector to be able to reach the identified gene region. A vector is an organism which is modified to carry the genetic code into the mutated region. Theoretically, this does not cause further illness or introduce viruses which induce ill health. Our first step in this therapy is to introduce copies of functional gene sequences into our chosen vector. This vector then acts as a template for the patient’s cells. Doctors must choose between stem cells and red blood cells based on the genetic condition being treated. This must must be done as the second step in the process is to collect some of the chosen cells from the patient’s body.
Now, we try and fuse these chosen cells with the functional gene, such that when placed back into the body, they are able to replicate. Before this though, we need to prepare our patient for chemotherapy so their body is ready to receive this functional package. Finally, we can infuse our patient with the gene-modified cells using intravenal injections. The doctors need to ensure that the grafting process is undertaken correctly and this means the patient must remain in hospital for a period of time.
Gene therapy can influence the variation in human populations. Biological process which are being manipulated have the potential of affecting the gene pool. This manipulation may result in unfavoured alleles becoming dominant within the human gene pool. On the other hand, recessive alleles may be selected which cannot handle current human selection pressures. Selected alleles by parents may result in the introduction of a wide range of gene expressions. Non-coding regions can manipulated which can result in unexpected phenotypes to appear which can not adapt to the selection pressures of the human environment resulting in ill health.
Gene therapy’s aim is to reduce the number of deaths related to mutated genes. There is an increased number of trials being conducted as technology advances and as a result the processes of gene therapy decreases in complexity. The graph displays that the number of trials corresponds to the global economic status. This can be identified after 2008 as there is a drastic a decrease in the number of trials being conducted due to the Global Financial Crisis.
Cancer is one of the most common illness trialed by gene therapy. Types of cancers targeted are gynaecological and nervous system. Monogenic diseases are second with 9.90% of the studies involving cystic fibrosis and Huntington’s disease. Lastly, around 8% of the trials focused on cardiovascular and infectious diseases.
Statistics show that Choroideremia Retinal disease trials has been receiving outstanding outcomes in regards of their improvements. The condition is underpinned by a radical vision loss that impacts primarily males. The first symptom of this condition is usually night vision impairment. These vision problem is caused by a constant loss of cells (atrophy) in the specialized light-sensitive tissue lining the back of the eye. Hence the results are showing a promising future for the treatment of the condition.
There are a variety of laws addressing the usage and techniques of gene therapy in order to reflect society as well as maintain the biological diversity of the human gene pool. All gene therapy studies in Australia must be aligned with the exemption programs for clinical studies established under the Therapeutic Goods Act 1989 (Clth) and supervised by the Therapeutic Goods Administration (TGA). The Gene Technology Act 2000 must also authorise the clinical trial, with the goal to protect people's health and safety and protect the environment by overseeing the dangers posed through gene technology by restricting certain GMO transactions.
There are certain strategies prohibited under Australian law to conduct clinical trials on germline cells outlined in the Prohibition of Human Cloning for Reproduction Act 2002 (Cloning Act). This restricts any modification on the human embryo outside the mother's body (s13), the insertion of any modified embryo into a woman's body(s20) and the alteration of any cell that can be inherited (s15).
I’d like to guide your attention to the ethical concerns regarding gene therapy. Ethics are defined as the moral grounds with which one chooses to govern themselves. A major ethical dilemma to consider is allowing doctors the ability to select certain characteristics deemed more desirable. This begs the question to whether we have the ability to select and alter the life of a child. For example, do the parents have the right to select for an offspring of high intellect? We must consider how modification affects the lives of offspring. This may cause ill health or change their life expectancy. Is it okay for us to make someone have down’s syndrome because their parents want a child that gives good hugs and smiles all the time? A recent ethical backlash was against Chinese biologist who practiced gene therapy on germline cells. The modification was the insertion of HIV virus into the zygote to create an offspring with HIV resistant genes. Although results of the trial is currently unavailable due to the unethicality of the trial. Biologist must consider the health effects when recruiting a viral vector to ensure there are no further ill health experienced. This safety concern questions the power of Biologist to introduce additional defective genes into patient's cells.
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