Researching Genetic Engineering and CRISPR-Cas9

 Sandra Mach, Verrazzano Class of 2021, completed major in Nursing 

Hi! My name is Sandra Mach and I am a Bachelor of Science registered nurse. My capstone project focuses on genetic technology, specifically CRISPR-Cas9, in the medical field.
 

Throughout my research, I realized that genetic technology is not only used in the medical field but also in our fruits and vegetables. To understand how gene-editing works, I had to research the basics and foundation of genes and genetic mutations. I learned that there are 23 pairs of chromosomes and any variation can lead to genetic mutation.

 Genetic mutation is defined as an alteration in the individual’s DNA sequence that permanently affects the gene ranging from a single base pair to a chromosome that includes multiple genes. There are seven types of possible mutations such as missense mutation, nonsense mutation, insertion, deletion, duplication, frameshift mutation, and repeat expansion. These variations include hair and skin color, height, shape, behavior, and risks to developing diseases. Furthermore, genetic variation influences societies’ ability to survive and reproduce. This is a great example of Darwinian evolutionary theory where it describes the mechanism of natural selection and “survival of the fittest”. Genetic variation that hinders survival and reproduction abilities are eliminated from the population. As a result, natural selection contributes to significant changes in the population.

Genetic engineering was first discovered and introduced in the 1970s to define the process of altering genetic composition of an organism via recombinant DNA technology. There are four recently discovered methods that are capable of breaking the DNA strand to create variations in the genome such as meganuclease (MN), zinc finger nuclease (ZFN), transcription activator-like effector nucleases (TALEN), and clustered regularly interspaced short palindromic repeats-CRISPR associated protein 9 (CRISPR-Cas9). CRISPR-Cas9 is a bacterial immune system where the single-strand RNA-guided Cas9 nuclease is linked to the targeted sequences to apply changes and allow direct correction of the disease-causing mutation. 

CRISPR-Cas9’s function can be explained in seven simple steps: (a) selecting an organism to manipulate, (b) selecting a gene or DNA sequence to manipulate, (c) selecting the CRISPR and gRNA, (d) constructing the gRNA or sgRNA by synthesis and cloning, (e) selecting the Cas9 protein, (f) delivering the sgRNA and Cas9 to the target cell, and (g) validating the manipulation.

 The CRISPR-Cas9 technique is often used to deactivate a gene pair and evaluate response to the medical treatment, specifically cells reacting to the medication/drugs. With the discovery of CRISPR-Cas9, it revolutionized the methodologies in hematology and oncology studies. This framework also provides the foundation in understanding the pathways similar to those found in cancer cells. It is evident that cancers are the result of mutations of the cell that lead to malignant phenotypes and CRISPR-Cas9 has the ability to target and correct these mutations. Furthermore, CRISPR-Cas9 also showed promising results for patients with sickle cell anemia and beta-thalassemia. To implement CRISPR-Cas9 technology, researchers must prove that the benefits outweigh the harm, it is in the patient’s best interest, and ethical by using evidence-based practice. 

Among all these revolutionary developments, there is serious ethical concern due to its ability to edit the entire genome. Any intentional or unintentional misuse of CRISPR-Cas9 technology can result in significant and unpredicted results on future generations and contribute to changes in the human gene pool. The scientific breakthrough has provided multiple medical treatment possibilities and brought awareness that there is a chance to cure diseases that were once believed to be incurable. CRISPR-Cas9 technology is the first step and a new beginning for future medical advancements.