The Science Journal of the Lander College of Arts and Sciences
Abstract
Transhumanism, designer babies, gene therapy, and super-soldiers are founded upon the same concept—genetic engineering. Clustered Regularly-Interspersed Short Palindromic Repeats (CRISPR) is a natural bacterial immune response method that takes advantage of gene manipulation to prevent an infection from mobile genetic elements. Since Mojica et al. (2005) first suggested the relationship between the CRISPR/Cas system and prokaryotic immunity, significant advancements have been made in understanding the mechanism and subsequent applications of CRISPR. CRISPR, has three main subtypes based on unique proteins and interference pathways and serves as an accurate and effective method for gene editing. Its mechanism consists of spacer acquisition, crRNA production, and interference. This highly dynamic form of genetic modification generates significant CRISPR sequence differences in species that are almost identical when comparing the rest of their genome. CRISPR/Cas9 demonstrates the simultaneous alteration of multiple gene loci in individual cells with a high degree of specificity and precision. Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disorder characterized by progressive muscle loss and eventual death in the late teens to early twenties. DMD affects calcium homeostasis, vasculature, genetic regulation, muscle movement, glycosylation, tissue remodeling, and inflammatory response mechanisms. Current treatments include antifibrotic pharmaceuticals, calcium maintenance, myostatin inhibitors, upregulation of uthrophin, nonsense suppression drugs, vector-mediated gene therapy, and cell transplantation. This review describes the mechanism of CRISPR/Cas9 and its application as a therapeutic approach to treating Duchenne muscular dystrophy.
Recommended Citation
Levitman, A. D. (2016). CRISPR/Cas9 as a Therapeutic Approach to Duchenne Muscular Dystrophy. The Science Journal of the Lander College of Arts and Sciences, 10(1). Retrieved from https://touroscholar.touro.edu/sjlcas/vol10/iss1/13
