Gene Therapy Essay Example

Major advancements within the past 50 years to our understanding and use of technologies, many have utilized it’s greatest potentials to help find a use in the biomedical world. With gene therapy now available to help counter genetic complications, humans are getting closer to overcoming nature and curing diseases that were otherwise thought impossible to do so. For hereditary diseases, CRISPR can target mutated genes within the embryo and remove the genes before it’s disastrous effects take place. A simple mutation to the DMD gene (the gene responsible for muscle contractions) can cause the life impending disease known as muscular dystrophy, but CRISPR can resolve this mutation with relative ease. A similar process can be done to fix genetic blindness, which is often due to one mutation that is easy to target and modify. Diseases that aren’t hereditary can also be fixed with gene therapy. Blood disorders that affect oxygen transportation can be cured by harvesting bone marrow stem cells from the patient and using CRISPR to make them produce fetal hemoglobin. Extraction of immune T-Cells and modification to the PD-1gene (a gene that creates the protein that tumors can bind to) can assist in helping patients overcome cancer. Gene therapy has even been useful in finding methods to prevent AIDS by using CRISPR to cut DNA of HIV and make them inactive or by using PCR to cultivate the CCR5 protein, a resistance to HIV found naturally in only a few individuals. 

Of course, gene therapy is not always going to be an easy fix. Some may wonder, after knowing that we can just produce CCR5 protein to prevent AIDS, why isn’t it widely used on everyone to make AIDS a problem of the past? To put simply, the main reason we are not utilizing this method is due to the high risk of dying younger. Why run the risk of dying at a younger age to prevent a possibility of contracting AIDS? There are also diseases that are much more complex and are due to multiple mutations within the gene. For example, CRISPR can be used to get to the origin and edit the CFTR gene to fix mutations that cause cystic fibrosis, however, there are multiple mutations and it is hard to target specific locations of the mutation. Huntington’s disease is a neurodegenerative disorder due to an abnormal repetition of certain DNA sequences and in most cases, doctors would be comfortable operating CRISPR to splice a similar mutation. However, with the location that the mutation for Huntington's disease is at, there is an enormous risk because any error can have tremendous consequences on the brain. Luckily, technology is evolving to help overcome these major obstacles and recently, the KamiCas9 was developed with a self destruct button to be paired with CRISPR in order to make it more precise. 

Surprisingly, disease prevention is not limited to gene therapy to humans either. Malaria is one of the leading causes to the human death toll and despite our best efforts, it is extremely difficult to provide resources or care to everyone, especially in developing nations. But what if, instead of curing those that already have been affected by malaria, we were to target the disease-carrying perpetrators themselves. Along with malaria, mosquitos have been responsible for transmitting other diseases like the dengue fever, yellow fever, and zika, and over one million people die from mosquito-borne diseases every year. With the ability to lay 300 eggs at a time, mosquitos are the perfect parasite host and almost impossible to eradicate. That’s why scientists have shifted their focus from trying to eliminate mosquitos and to changing the types that transmit diseases. Using genetic engineering, scientists have created a strain of mosquitos that are immune to malaria by inserting an antibody gene that targets malaria. These mosquitos will never spread malaria and by making the gene a dominant trait, “99.5% of all engineered mosquitoes offsprings carry the antimalarial agent” (Kurgazat, “Genetic Engineering and Diseases). Releasing enough of these engineered mosquitoes into the wild to mate with normal mosquitos will allow the malaria blocking gene to spread rapidly and scientists believe the change would happen so fast that malaria will not be able to adapt to the change quick enough. With this, malaria may become extinct and if all goes to plan, this may just be the beginning of curing multitudes of parasite-borne diseases. Different mosquitoes carry zika and dengue fever, ticks transfer lyme disease, flies transmit sleeping sickness, and fleas transmit the plague. Implementing this technique could save millions, prevent suffering, and overall benefit the human race on an unbelieve scale. So why haven’t we used it yet?