Gene Therapy Treatments for Genetic Diseases Although Gene therapy is being studied and used to treat genetic diseases, it is not quite ready for world wide use.

Glossary

Vectors - The viruses or pathogens that are used in gene therapy to carry the treatment and correcting DNA.

Genes - Characteristics passed on from parents to offspring through genes that are in DNA.

DNA - Self replicating cells that carry the genes and the instructions for the body to make more cells.

Mutation - A disorder in DNA where the DNA is not replicating correctly and causes some health problems like genetic diseases.

Somatic gene therapy - The type of gene therapy where the benefits only effect the person receiving the treatments.

Germ line gene therapy - The type of gene therapy where the benefits of the treatment effect the patient and the generations after.

How does gene therapy work?

Gene therapy looks to correct mutated genes in the patient by replacing or killing the bad genes. In the article "How does gene therapy work?", the U.S Department of Health & Human Services state, “If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.” They use special, more advanced medical technology to fix the genes in the patient. The most common way of introducing the new genes is through viruses or vaccines; Thomas, Ehrhardt, and Kay state, “The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes” (346). Vaccines are used because it is the most efficient way to get the correcting genes in the patient through the use of the vectors to get it inside the DNA. There are many risks to using gene therapy as well.

What are the risks of using gene therapy?

Gene therapy is the most effective known genetic disease treatment. The treatment is not fully complete and safe yet however; gene therapy researchers and developers Thomas, Ehrhardt and Kay state, “The inappropriate activation of inflammatory responses can be highly dangerous; a massive systemic inflammatory response that was induced by an adenovirus vector led to fever, disseminated intravascular coagulation, multiorgan failure and the eventual death of a patient during a 1999 trial for ornithine transcarbamylase (OTC) deficiency.” Using gene therapy can side effects for the patient as well. A statistic from the Animal Cell Technology Industrial Platform in the article, "Gene Therapy", says that during a gene therapy experiment, 10 out 11 patients experienced toxicity from the treatment. Multiple types of gene therapy is used also, including germline and somatic gene therapy.

Why is germline gene therapy different from the more common used somatic gene therapy?

Germline gene therapy is different from somatic gene therapy in one specific way. The effects of germline gene therapy are passed on to the patient's offspring. Henry Miller, former FDA worker, states, “Somatic cells are any cells in the body except eggs or sperm, so modifications in them are not heritable— that is, passed onto offspring.” The effects are also passed on through all generations of the person who is treated, the Wellcome Genome Campus, in the article, "Is Germline Gene Therapy Ethical", states, “Germline gene therapy enables the correction of disease-causing mutations that are certain to be passed on from generation to generation, sparing future generations from suffering from the disease.” This type of gene therapy is different from somatic gene therapy since the effects get passed on to the patient’s offspring and the generations after. This type of gene therapy is still being studied and is not yet used regularly.

How long will it take until gene therapy can be used regularly?

Gene therapy is making a lot of progress towards being able to be used regularly for genetic diseases. A study from the University College London, in the article, "Gene Therapy: Progress and Predictions", states, “We predict that the next 25 years will see improvements in safety, efficacy and manufacture of gene delivery vectors and introduction of gene-editing technologies to the clinic.” Gene therapy will be able to be used in hospitals and it will be much safer and more efficient within the next 25 years. Gene therapy trials are also getting further along in the stages, according to a statistic from New Big Future Inc. in the article, "Gene Therapy Progress is Summarized", about 5 percent of 2,000 gene therapy trials in 2013 reached phase III of the treatment. Although gene therapy is not the only option for treating genetic diseases, it is the most effective known treatment.

What are alternatives to gene therapy?

With the risks that come with gene therapy, there are new alternatives that are being looked at to treat genetic diseases. The International Rare Disease Research Consortium states in the article, "Chemical drugs could be an alternative to gene therapy to treat certain genetic diseases", “In addition or as an alternative to gene therapy, chemical drugs could offer benefits to treat certain genetic disorders resulting from loss-of-function (LOF) or gain-of-function (GOF) mutations.” Some chemical drugs are being developed and tested to be an alternative treatment to gene therapy. Also, scientists are looking at medical procedures rather than drugs. Fulvio Mavilio, a gene therapy researcher, states, “A variety of strategies have been developed to increase HR efficiency, such as the use of site-directed nucleases to introduce double-stranded cuts at the target site.” This is another method they are looking to use to treat genetic diseases.

This TED talk is explaining more about gene therapy and how it works. Also, it explains why it is relevant and important that gene therapy is studied and what use it has.

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