Essay Sample on CRISPR: A Potential Alternative to Gene Therapy

Introduction

Some people might argue that CRISPR is another cheaper option and possibly better than gene therapy. Notably, this technology has been used by many researchers and scientists to show how beneficial it is to treating diseases and reversing some of the fundamental genetic issues human beings undergo. CRISPR is a technique that employs gene editing, where a specific genome is targeted (Pged, 2020). Clustered regularly Interspaced Shorts Palindromic Repeat (CRISPR) uses the RNA and nuclease (Cas9) to alter the genetic coding of cells where the incorporated genome sequence is used to replace the existing sequence removing the ailment such as cancer among other diseases. However, this process resembles gene modification, which has brought controversies in medicine’s ethical considerations for the use of CRISPR. This technique alters the genes of an individual forever, and the altered sequence would be transferred to the offspring (Pged, 2020). Gene therapy is the perfect choice because its effect is not transferable to offspring.

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Gene Therapy

How often have we heard people losing lives to chronic diseases that have been prevalent since the 1980s or even past that? How many lives have been lost to diseases that can be solved through genetics? How many people are suffering due to ailments that seem to be impossible to find the cure for? All these questions could and can be answered through genetics. Genetics is key to unlocking unlimited potential in the field of medicine. Imagine a world where there are no related genetic diseases (Mohammed, 2015). Imagine a world where ailments in which specific genes are causes are eliminated for the good of humanity. Therefore, all this is possible with the right research and the right effort. Gene therapy is the answer to all these questions.

Gene therapy could be the answer to cancer, and it could be the answer to leukemia and other chronic diseases that have taken so many lives. Since 1990, over 2000 clinical studies have been conducted and gained approval globally when the first gene therapy trials were done. Improvements are on the rise as many scientists have devoted their lives to unlocking some of the mysteries in genetic code and sequencing to help people overcome some chronic ailments. Researchers sought to treat genetic and genetically acquired illnesses without traditional treatment methods (Mohammed, 2015). Ailments targeted by traditional dynamic methods promise a medical approach: sickle cell anemia, thalassemia, cancer, cardiovascular disease, infectious diseases, and single monogenic disorders.

Why Gene Therapy?

Firstly, gene therapy is a technique that is experimental based on DNA insertion to cells of organisms so that a disease could either be treated or its progress slowed down, especially to ailments that emanate from mutation of one or more genes. Genetics presents various ways in which gene therapy could be achieved. They include removal of mutated genes with a copy of a healthy gene, removing the function of a gene that is defective that causes the disorder (knockout) and knockin where a new gene is inserted into the body to fight diseases. This technique aims at treating diseases at the DNA level as opposed to other traditional forms of treatment.

Business Atmosphere and the Future of Gene Therapy

Since the death of Jesse Gelsinger, one of the dark past related to gene therapy, there have been tremendous improvements in the field. Better clinical and scientific understanding has been established, making gene therapy a success over the years. Scientists and researchers have been more conversant with immunogenicity and integration patterns of viral vectors and modification to delivery mechanisms, albeit technological advances. In 2018, at least 150 investigational new drugs were filed for gene therapy, which is an indication that the market for gene therapy is promising, and the future is brighter (Carpa et al., 2019). According to Mckinsey & company, growth is thought to have stemmed from ASOs and RNAi, where Spinraza and Onpattro are the initial therapies of this kind, deploying AAV and lentivirus therapies like Zynteglo. Moreover, through accelerated regulatory review pathway where the approval process is expedited, most of the gene therapies have risen to market rather quickly (Carpa et al., 2019). This theory is highly attributed to the larger number of patients affected by diseases that gene therapy could offer.

Conclusion

Most people relate gene therapy to riskiness and danger with insertions on invitations to discrimination to disabled people. The point of being a dangerous technique is often attributed to the fatal deaths resulting from gene therapy clinical trials. However, no clear statistics as to what extent gene therapy played in some of these scenarios and whether gene therapy was the sole cause for the fatalities. Moreover, arguments revolve around the fact that through the delivery mechanisms employed in gene therapy; vectors might deliver the DNA to other cells other than the target cells resulting in unforeseen results. The application of viruses as vectors might cause disease. These viruses may fail to adhere to the assumption that they are innocuous, escaping the immune response and ensuring the new gene isn’t the cause for disruption of other genes.

References

Carpa, E., Smith, J., Yang, G. (2019, October 2). Gene therapy coming of age: Opportunities and challenges to getting ahead. McKinsey
https://www.mckinsey.com/industries/pharmaceuticals-and-medical-products/our-insights/gene-therapy-coming-of-age-opportunities-and-challenges-to-getting-ahead

Genetic Home Reference. (2020, April 12). What is gene therapy? Genetic Home Reference. https://ghr.nlm.nih.gov/primer/therapy/genetherapy#:~:text=Gene%20therapy%20is%20an%20experimental,of%20using%20drugs%20or%20surgery.

Mohammed S, W. (2015, June 11). Gene Therapy as an Alternative Treatment for Both Genetic and Acquired Diseases. ResearchGate. https://www.researchgate.net/publication/314299250_Gene_Therapy_as_an_Alternative_Treatment_for_Both_Genetic_and_Acquired_Diseases

Pged. (2020, March 23). Genetic Modification, Genome Editing, and CRISPR. Personal Genetics Education Project. https://pged.org/genetic-modification-genome-editing-and-crispr/