Introduction: What is Gene Therapy?

Gene therapy is a technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder (e.g cystic fibrosis, a genetic condition that causes a build-up of mucus in the lungs causing breathing problems, muscular dystrophy, the weakening of muscles over time caused by a disease)  by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing many different  approaches to gene therapy, including:

●         Replacing a mutated gene that causes disease with a healthy copy of the gene by cutting and pasting with enzymes.

●         By essentially muting or deleting a mutated gene that can cause or is causing problems to the patient.

●         Producing a new gene that can improve immune responses to diseases.

(U.S national library of medicine, 2019)

In this essay, I will be discussing what gene therapy is in more detail the impact gene therapy has now and how big of an impact it can have in the future.

What is Gene Therapy

Gene therapy is designed to put genes into cells to make up for abnormal genes or to make a protein that will give benefits. If a mutated gene causes an important gene to become dysfunctional, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.

A gene that is inserted directly into a cell usually does not function. Instead, a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can take the new gene by infecting the cell. The viruses are modified so they can’t cause problems and infections when used in people. Some types of viruses, such as retroviruses, insert their genetic material into a chromosome in the cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome.

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The vector can be inserted via injections or given endovenous directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient’s cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein.

(U.S national library of medicine, 2019)

Figure 1: Diagram to illustrate the process of gene therapy

(bioninja)

Gene Therapy: Past

The first idea of incorporating new genetic functions in mammalian cells in the late 1960s they did this by inserting the gene with a micropipette directly to the mammal cell another way they did this way by exposing the cells to a DNA precipitate containing the desired gene for the mammal cells

(What is Biotechnology, 2019) 

Patrice Denefel (2011, p.27) Martin Cline made a first early attempt of human gene therapy in 1979 by trying to treat severe thalassemia through an ex vivo beta-globing gene transfer protocol in the bone marrow on two patients however the protocol was not accepted/approved by the regulatory board so the study was terminated and Cline had to resign. It wasn’t accepted as gene therapy was very premature at the time.

(National Cancer Institute,1990) R. Michael Blaese, W. French Anderson, and Kenneth Culver announced the start of the first gene therapy trial for treating children with severe combined immunodeficiency.

However, with clinical trials taking place, the hopes were thrown out the window when the death of the first patient took place in 1999.

The first idea of incorporating new genetic functions in mammalian cells in the late 1960s they did this by inserting the gene with a micropipette directly to the mammal cell another way they did this way by exposing the cells to a DNA precipitate containing the desired gene for the mammal cells

(What is Biotechnology, 2019) 

Gene Therapy: Present

After a couple years of failures and a number of cases of unsuccessful treatment and failed trails after a decade more safe and effective vectors arrived and in 2007 Jean Bennett demonstrated a small trial that gene therapy could be promising for an inherited retinal disease and in 2015 trials took place which showed that the therapy could also help hemophilic patient who no longer need to take blood clotting factor drugs. More good news also arrived in 2015 from trials of gene therapy for thalassemia and sickle cell anemia that those affected can survive and be healthy without blood transfusions due to gene therapy. In 2016 Europe licensed a second gene therapy, developed by GlaxoSmithKline for children affected by ADA-SCID.

Figure 2: a graph to show how the effect gene therapy has on haemophilia B

At the end of 2016, a group of Chinese scientists Launched a safety trial to see if it was possible to treat cancer patients by using CRISPR-Cas to disable a particular gene in their cells that codes for a PD1 protein which holds back a cell’s immune response to cancer cells.

(Lara Marks, Royal Society of Chemistry, 2017) Although in recent years scientists have made strides of development in gene therapy there are still some issues that need to be looked at in terms of the safety of the whole procedure. The biggest difference between gene therapy and conventional drugs and biological products is that gene therapy is difficult to stop the treatment once it has been given and we still don’t know the nature and the l long term risk the therapy it can cause on our bodies. Also, gene therapy is expensive, sky-high prices due to the therapy being custom made for individuals

Patrice Denefel (2011,p.40) Gene therapy is now understood to be difficult for the clinical development field and there’s still a trend of private investors staying away from this area.

The driving force has remained often too long in the hands of academic research and because of this clinical development has been failing because of translations of the research to the lab

Finally, although viral vectors are considered the best in class to achieve efficacy in men there are major adverse effects such as vector related oncogenesis in some trials and complex immune responses

Gene Therapy: Future

In the future Gene therapy will be very useful to tackle and take on inherited diseases that right now we can do anything about as the medication isn’t as useful. But as listed up above we can’t there are many issues with gene therapy that need to be overcome.

Gene therapy also has the potential to cure cancer by silencing a gene.

However gene therapy can be used for unethical things such as “designer babies” designer babies are made by the parent selecting favoured genes for the baby so they can choose what colour eyes the baby has or what colour hair however manipulating the genes may not be so bad as we could possibly get rid of the chance of the baby being a cystic fibrosis carrier and if all babies get this treatment to not be carriers of inherited diseases the next generation can grow up with less chance of being affected or even being a gene carrier Although this is good manipulating the genes for appearance is unethical as it increases discrimination and bullying towards the children as some features are “favoured”

In conclusion gene therapy has a lot of work to be done on and has time to improve however someone needs to fund the research also it has to be used for the right things like eradicating genetic diseases like cystic fibrosis, thalassemia, Tay-Sachs and Sickle Cell Anemia to name a few. But before that, we need to know the side effects and long term effects gene therapy seems like an easy concept but it’s hard to execute with many failed trials however Gene therapy is an exciting new approach that is just making the news.

In the future, this technique, if successful, could be the treatment we can all turn to for survival and it will become very common around the world. Gene therapy will change the field of medicine forever as scientists research and discover more into genes and their functions, the potential of this treatment is limitless. The genome is the blueprint of the body this could potentially huge step to changing the blueprint and becoming stronger and healthier humans however the key to our future is locked in our genome. As researchers start to understand this blueprint and try to find the key, our lives will be forever changed. The next couple of steps into this research and trials will be make or break either it was a good idea and too good to be true or scientists make a groundbreaking breakthrough and hopefully will lead to a better society to live in.

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