How the CRISPR-Cas9 Gene Editing Complex Can Be Used to Treat Human Immunodeficiency Virus 1 and Sickle Cell Disease with Immune Cell Therapies and Genetic Engineering with Insight Into its Societal Impacts

Using CRISPR/Cas9 gene editing in modern medical sciences to develop treatments for genetic diseases like HIV-1 and SCD

Abstract

HIV affects approximately 38 million people in the world today, according to hiv.gov, and Sickle Cell Disease affects an estimated 1 to 3 million people worldwide (U.S. Department of Health & Human Services, 2021). Today, modern drugs used to treat HIV-1 make living with it more manageable, however, this is not the case with Sickle Cell Disease. No effective treatments are available to provide a cure, but CRISPR has provided new research opportunities that have been proven effective in treating these diseases. This paper highlights the use and effectiveness of both the CRISPR gene editing system and the processes of experimentation in multiple fields. More specifically, researchers focus on the effectiveness and potential for future treatments for gene editing in inherited diseases like Sickle Cell Disease, and contagious diseases like Human Immunodeficiency Virus 1 (HIV-1). The potential of CRISPR-Cas9 is paramount in discovering gene-editing methods within these diseases, by allowing excision of the RNA and genomic DNA to delete, replace, and discover new vulnerabilities to guide effective treatment. This paper seeks to explain how the biochemical applications of the CRISPR-Cas9 protein complex can be used in genetic engineering to treat HIV-1 and Sickle cell disease (SCD), using immune cell therapies and other treatments. It can be supported and discussed within the context of previous gene-editing technologies and where we stand with future technological development. This can provide insights into social development if given the resources to alter the attributes of humans and the natural world.

Keywords: CRISPR, Cas9, HIV-1, SCD, Hematopoietic Stem cells, Germline, Somatic, HDR, NHEJ, mutation, PAM, Reverse Transcription