Abstract for:CRISPR/Cas9 Gene Editing System and the Efficacy Challenges

Gene editing is an exciting and controversial field. There is hope for advances in diseases such as cancer, HIV/AIDS, and genetic disorders. CRISPR is widely considered the most precise and most cost-effective way to edit genes. There yet remains guidelines for designing preclinical trials studies and ethical issues surrounding gene editing. One of the most important challenges in this field is the ethical issues regarding the application of the CRISPR/Cas9 system in relation to gene therapy. Ethical issues include problems of safety, setups in clinical trials, and the criteria for including patients for research and treatment. Other challenges and issues include specificity to ensure the correct targeted gene can be reached. Ethical concerns regarding gene editing in embryos are also rising. Safety concerns and lack of guidelines remain as other concerns. A framework that includes a set of guidelines and code of ethics needs to be developed to guide scientists through the process of conducting their research. This work explores the major issues and challenges that effect the efficiency of CRISPR/Cas9 systems. A system dynamics modeling approach is proposed to tackle the different variables that would have an impact on the efficacy and provide a better decision factor for scientists.