Essay Sample on Genetic Engineering: Eradicating Sickle Cell Disease?

Introduction

The issue I have chosen to research in the natural sciences is Genetic Engineering. Genetic engineering, in its simplest terms, is the purposeful adjustment or alteration of an organism's DNA in order to improve specific characteristics or traits (Laney, 2018). I specifically want to know if humans should take advantage of CRISPR-Cas9 technology to eliminate sickle cell disease? Sickle cell disease affects over 100,000 Americans and millions around the world (Powars, 2019). It is caused by a variation in the B-globin gene, one of the genes that help produce hemoglobin (Powars, 2019). When looking at these blood cells, they are abnormally shaped, and symptoms include severe pain and swelling in the extremities and risk of stroke or bacterial infections (Powars, 2019). CRISPR-Cas9 is an efficient and precise technology that allows scientists to target, delete and repair any mutated sequence of DNA in any gene (Shwartz, n.d.). There is currently no cure for sickle cell disease and the only treatment options that existed before the CRISPR-Cas9 technology was bone marrow transplants and blood transfusions (Powars, 2019). The CRISPR technology isn't perfect, but research by Porteus suggests that it doesn't need to be (Shwartz, n.d.). Symptoms of sickle cell disease only occur when the proportion of sickled cells in the bloodstream is above 30 percent (Shwartz, n.d.). If 70 percent of the red blood cells are healthy then the patient can be symptom free (Shwartz, n.d.). Porteous believes that if 20 percent of stem cells are corrected in the bone marrow, then it will be most likely put patients above the 70 percent threshold because healthy red blood cells live 5 times longer than diseased cells and quickly outnumber them (Shwartz, n.d.).

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My Question

Should humans take advantage of CRISPR-Cas9 technology to eliminate sickle cell disease?

My Science Resources

The name of my first article is "CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells." The genome-editing technology may be used in correcting sickle cell mutation of the hemoglobin gene in the hematopoietic cells. These are the fountain for blood cells, which circulate in the body. Such correction enhances the production of the red blood cell, which synthesizes the hemoglobin. The genome tools such as CRISPR/Cas9, Transcription Activator-like Effector Nucleases (restriction enzymes that can be made to cut specific sequence of DNA), and Zinc Finger Nucleases (DNA binding proteins) are powerful genome editing technologies that have the therapeutic ability for sickle cell disease and other disorders. Such technological tools are being used in clinics, and health providers are exploring their ability to correct genes for sickle cell disease (Hoban et al., 2016). More specifically, through in vitro, correction rates in bone marrow CD34+ cells from sickle cell disease patients using CRISPR averaged 20% and led to the production of corrected hemoglobin A at the rates of 7% (Hoban et al., 2016). These were similar to the rates already reported using ZFNs (Hoban et al., 2016). If the correction rates presented here could be maintained long term, repopulating hematopoietic stem cells, the potential for clinical benefit is high (Hoban et al., 2016).

The name of my second article is "Therapeutic Crispr/Cas9 Genome Editing for Treating Sickle Cell Disease." Sickle cell is among the common single-gene disorders which affect several people in the world. The disease is caused by mutation of the hemoglobin. Nucleotide replacement (the mutation which alerts the amino acid) of the amino acid that changes glutamic acid (amino acid that contains proteins) to valine (amino acid with excess protein) may result in the formation of sickle cell, which weakens the performance of the red blood cell. The article seeks to determine the capability of the genome editing in providing a permanent cure for sickle cell disease through clinically correcting the mutation. The study designed CRISPR/Cas9 and a donor template, which was used for introducing the sickle mutation into hemoglobin in the blood cell. In assessing the result of the genome editing in curing sickle cell that was introduced by CRISPR/Cas9 system, the study developed a novel digital droplet PCR that was used to measure the degree of homology repair (gene conversion in which no information is lost in the process) and non-homologous end joining (pathway for repairing DNA double-strand break). Homology is the presence of similar bases in related genes. The study found that the efficacy in correcting the site-specific gene can be enhanced through the optimization of the system for genome editing. Determining the presence in the genetic make-up of every person was relatively high and could be of two identical or different alleles (Park, Lee, Deshmukh, & Bao, 2016). More imperatively, the gene in the corrected cells is capable of differentiating into erythroid (cell charged with transportation of oxygen), which produces hemoglobin. The result demonstrated proper evidence which can be used for clinical translation in the correction of the gene for providing treatment to patients with sickle cell disease.

My Audience and Message

My audience is those who have sickle cell disease or know of someone who has sickle cell disease and is looking for a new treatment that would potentially take away their symptoms for good. Vertex and CRISPR Therapeutics have co-developed CTX001 as CRISPR Cas9 edited treatment for Sickle Cell Disease and Thalassemia (ENP Newswire, 2017). Although this treatment is still in the beginning stages and is an investigational gene editing treatment, the preclinical data presented about CTX001 showed that there were increases in fetal hemoglobin (ENP Newswire, 2017). The reactivation of fetal hemoglobin is important to patients with sickle cell disease because it inhibits the multiplication of the sickle hemoglobin and the resulting symptoms (Fathallah & Atweh). The way fetal hemoglobin does this is by providing an abundant amount of oxygen to the patient's red blood cells, keeping the patient's defective hemoglobin from activating (Weintraub, 2016). The elevation of fetal hemoglobin by CTX001 has the potential to eliminate the symptoms for people who have Sickle Cell Disease (ENP Newswire, 2017).

Identify Principles

The natural science principle that applies to my topic is Genetics. At its most basic, genetics denotes the study of hereditary, identifying the fundamental roles played by DNA sequence in instructing cell functioning; DNA performs cell operations through chemical processes primarily through the production of proteins. Genes are the fundamental units of hereditary and denote the basic traits passed from generation to generation or from parents to offspring. According to Arduengo (2019) research, genetic variation is a significant subject in all fields of Biology as it is identified as the key driver of evolution by natural selection.

Explain Principles

Sickle cell disease affects over 100,000 Americans and millions worldwide and is a painful inherited condition. It is inherited when a person receives two genes that carry the sickle cell hemoglobin from their parents (Harvard, 2002). Conversely, the parents of the autosome recessive individual carry a copy of the mutated gene each but do not express the signs and symptoms. Less oxygen in sickle cell hemoglobin after its released in the body tissues makes them crescent-shaped, rigid and sticky. Also, they tend to occlude small blood vessels, causing painful ischemia. The sickle cell disease is referred to as a recessive condition as an individual must have two copies of the mutated genes for the disorder to be expressed. Using a genetic engineering technology called CRISPR-Cas9 could offer a safe and efficient way to repair the genetic mutation that causes this disease. CRISPR-Cas9 denotes a unique advanced technology developed to enable professional personnel such as medical researchers to edit parts of a genome by removing adding or altering sections of the DNA sequence.

My Hypothesis and Experiment

My hypothesis would be that CRISPR-Cas9 technology will effectively eliminate and cure sickle cell disease. To test this hypothesis, I would get qualified geneticists to look at cell cultures that contained the sickle mutation where they would then modify and edit the B-globin gene with CRISPR-Cas9 ("Sickle Cell Disease | National Heart, Lung, and Blood Institute (NHLBI)," 2019). Once repaired, the modified stem cells with the sickle-cell trait would be injected back into the test subject's bloodstream where, ideally, they would be creating millions of healthy red blood cells. This could potentially make the cure for sickle cell disease available around the world, primarily because it corrects the sickle mutation with a person's own gene-corrected stem cells.

From Professor about "My Hypothesis and Experiment"

CRISPR experiment is vital and significant. To set up the experiment, consideration of the three CRISPR steps; design an optimum guide RNA and other components or the experiment, genome engineering using the CRISPR technology and analyzing the effectiveness of the created components is important ("How To Use CRISPR," 2019). Conversely, bone marrow biopsy can be used to harvest the hematopoietic stem cells from sickle cell patients. The setup experiment involves a design CRISPR Guide, and use of the proper tool to introduce CRISPR components. The experiment measures include considering the on-targets and off-targets potential of the RNAs. The effectiveness of the experiment can be determined by establishing the proportion of sickled red cells and normal cells of the patients over a period of time. The patient needs to be closely monitored within the period.

Resources

https://sickle.bwh.harvard.edu/scd_background.htmlReferences

Arduengo, M. (2019). Genetics. Salem Press Encyclopedia of Science. Retrieved from

https://search-ebscohost-com.ezproxy.snhu.edu/login.aspx?direct=true&db=ers&AN=88833240&site=eds-live&scope=site

Fathallah, H., & Atweh, G. F. (2006). Induction of fetal hemoglobin in the treatment of sickle

cell disease. Hematology. American Society Of Hematology. Education Program, 58-62. Retrieved from https://search-ebscohost-com.ezproxy.snhu.edu/login.aspx?direct=true&db=cmedm&AN=17124041&site=eds-live&scope=site

Hoban, M. D., Lumaquin, D., Kuo, C. Y., Romero, Z., Long, J., Ho, M., ... Kohn, D. B. (2016).

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells. Molecular therapy : the journal of the American Society of Gene Therapy, 24(9), 1561-1569. doi:10.1038/mt.2016.148

How To Use CRISPR: Your Guide to Successful Genome Engineering. (2019). Retrieved October 13, 2019, from https://www.synthego.com/guide/how-to-use-crispr

Laney, D. A. . M. S. . C. G. C. . C. C. R. C. (2018). Genetic Engineering. Salem Press

Encyclopedia of Science. Retrieved from https://search-ebscohost-com.ezproxy.snhu.edu/login.aspx?direct=true&db=ers&AN=89250470&site=eds-live&scope=site

Park, S. H., Lee, C. M., Deshmukh, H., & Bao, G. (2016). Therapeutic Crispr/Cas9 Genome Editing for Treating Sickle Cell Disease. Blood, 128(22),4703. Accessed September 19, 2019. Retrieved from http://www.bloodjournal.org/content/128/22/4703

Powars, D., M. D. (2019). Sickle cell disease. Magill's Medical Guide (Online Edition).

Retrieved from https://search-ebscohost com.ezproxy.snhu.edu/login.aspx?direct=true&db=ers&AN=86196333&site=eds-live&scope=site

Sickle Cell Disease | National Heart, Lung, and Blood Institute (NHLBI). (2019). Retrieved October 13, 2019, from https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease

Shwartz, M. (n.d.). Target, delete, repair: CRISPR is a revo...