A relatively new technology known as CRISPRCas9 has the capability to create global impact upon the way scientists investigate and medical professionals treat certain diseases in the future. ‘CRISPR’ which stands for ‘clusters of regularly interspaced short palindromic repeats’, palindromic meaning that it reads the same both forwards and backwards is a special kind of DNA found in bacteria (U.S. National Library of Medicine. 2020) .
Unique characteristics of CRISPR allow viruses that previously attacked the organism to be stored within ‘memory banks’ to fight off future ‘attacks’. When used in association with a Cas9 protein which is an enzyme (speeds up the rate of chemical reactions within the cell) that cuts foreign DNA, scientists can edit DNA sequences within living cells. This technology thus has the potential to correct various genetic defects, treat disease and improve crops; with a simple programmable genome editing tool.
By applying CRISPR technology to immunodeficiencies such as by providing treatment to Chronic Granulomatous Disease (CGD), over 6 million individuals globally with an immunodeficiency can seek long-term treatment (British Society for Immunology. 2017) . Immunodeficiencies ultimately result in a full or partial impairment of the immune system by genetic defects or environmental factors with up to ‘50% of the developing world’ being affected by malnutrition-related secondary immunodeficiencies (result of illness or external environmental factors).
CGD is an ‘inherited primary immunodeficiency’ where victims are vulnerable to life threatening infections due to a defect mutation (an alteration to the nucleotide sequence of a genome) in the NOX2 protein of the immune system that destroy potential infectious bacteria. Similar to other immunodeficiencies, CGD is hereditary meaning it is transmitted from one generation to the next, consequently, by using CRISPR technology the defect mutation can be resolved permanently to avoid the inherited disease from continuing to the following generation as well as treating the current patient. Thus, this technology has the capability to remove immunodeficiencies to ultimately improve the quality of life and reduce the risk of life-threatening infections from ‘infiltrating’ the immune system (Paediatric Clinics of North America. 2020) .
Yet some may argue upon the ethical viability of generating heritable changes to the human genome.
Further research has reflected the effectiveness of CRISPR technology in eliminating the Human Immunodeficiency Virus (HIV-1/AIDS) in animal models through intravenously (administered into a vein) injecting a ‘multiplex of single-guide RNAs and Cas9 (Molecular Therapy. 2018) . Within mice, this saw a significant reduction in HIV-1 expression in several of their organs and tissues. This study thus reflects the potential step toward human clinical trials and the capability to treat HIV-1 on a broader scale. In 2019, 44 million individuals were known to have HIV-1 which attacks the body’s immune system by destroying CD4 cells (white blood cells) and the disease killed 1.1 million (UNAIDS. 2019) .
Despite these advancements, the largest stakeholder of HIV-1 CRISPR technology currently lacks access to hygiene and medical resources to apply the treatment. Developing nations often have a pronounced epidemic of the virus due to poverty, a low level of education and limited medical resources as reflected within Eastern and southern Africa where 20.6 million people live with HIV-1 (Virol Journal. 2019) . Yet, due to financial and geographical disadvantages; this majority will be potentially unable to access CRISPR technology as treatment within the future. Consequently, with the support of governments and regulations placed upon pharmaceutical companies a low-cost method of accessing the technology could be developed to provide fair access to these stakeholders that currently lack equal treatment.
However, the possibility of ‘off targets’ and mosaic mutations (presence of more than one genotype in an individual) when applying CRISPR technology upon immunodeficiencies threatens the safety and health of already predisposed vulnerable patients where the risk of infection can be life-threatening (Science Direct. 2019) . Further, within developing nations where medical resources are not at a sufficient standard these risks can be more pronounced as there is possibility that it could provide further threat to their immunodeficiency condition.
Amongst this, recent studies have discovered ‘serious problems’ with CRISPR as they demonstrated that CRISPR altered cells have the potential to lose key anti-cancer mechanisms and consequently increasing the risk of the cells initiating cancerous tumors (Nature Medicine. 2020) . A study induced that Cas9 enzymes have potential toxicity to kill human pluripotent stem cells (hPSCs) (master cells that produce cells and tissues for repair) and consequently cell replacement therapies using CRISPR-Cas9 technology should ‘proceed with caution’ (Boston Children’s Hospital. 2020) .
Correspondingly, CRISPR technology will cause a significant removal of defect mutations within genes, however, by altering the genome of an extensive quantity of individuals such as treating all patients with HIV-1 or CGD a strong impact upon human biodiversity will occur.
“It seems Gene Editing is going to eliminate all disease” – “Or kill every last one of us”
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