Preprint / Version 1

Gene Editing and Healthcare Applications

An outline of how CRISPR-Cas9 serves to advance healthcare


  • Sanika Kathale Cumberland Valley High School



CRISPR-Cas9, Gene-editing, Genetics, Genetic engineering, Bioethics, Sickle Cell Anemia, Cystic Fibrosis, Gene Research


The advances in the field of genetics and gene editing have led to fascinating applications of gene modification technology in the work of healthcare research. Genetic mutations and genetic diseases can be impacted by the therapies and treatments that have progressed due to CRISPR-Cas 9. CRISPR-Cas9 consists of a guide RNA (gRNA) which contains a section of the target DNA and an endonuclease that cuts DNA. CRISPR-Cas9 is great at knocking out gene expression and especially useful for autosomal dominant disorders because if CRISPR-Cas9 disrupts the function of a dominant, disease-causing allele, only the recessive allele is expressed (Kathleen et al. 2020). It is imperative to keep in mind gene therapy progression as well. Gene therapy is the oldest form of genetic medicine and is based on gene transfer technology, such as insulin bacteria. Insulin bacteria are the result of the human insulin gene being inserted into a bacterial plasmid. DNA can be delivered by a virus shell by inserting the appropriate replacement DNA (Petrich 2020). All forms of gene therapy in humans must undergo clinical trials in order for treatments to be approved by the FDA. Clinical trials must also follow criteria that all involved must adhere to: trials must have strict eligibility criteria, researchers must communicate about serious adverse events in other participants, and participants must show informed consent (Gupta 2013). Lastly, because tempering with genetic material can be such a risky method for healthcare professionals to resort to, ethical and safety considerations of using gene editing for human health and disease must be considered. Overall, how genetic mutations cause diseases such as cystic fibrosis and sickle cell anemia, how CRISPR tools can be used to combat it, and what the bioethical considerations are of using gene editing treatments can all be better understood through gene editing research.


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