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Disease Prevention

CRISPR technology is a promising tool not only for genetic disease corrections but also for the prevention of viral and bacterial infections. Utilizing CRISPR–Cas therapies, researchers have targeted viral infections like HSV-1, EBV, HIV-1, HBV, HPV, and HCV, with ongoing clinical trials for an HIV-clearing strategy named EBT-101. Additionally, CRISPR has demonstrated efficacy in preventing viral infections such as IAV and SARS-CoV-2 by targeting viral RNA genomes with Cas13d, and it has been used to sensitize antibiotic-resistant S. aureus to treatment through Cas9 delivered via bacteriophages.

Despite existing evidence of CRISPR technology, advancements in the field persist in reducing limitations. Researchers developed a new, gentle gene editing method for embryos using nanoparticles and peptide nucleic acids (PNAs). Delivering editing tools without harsh injections, the method successfully corrected genes in mice without harming development. While ethical and technical questions remain, this study paves the way for potential future use in improving livestock and research animals, and maybe even in human embryos for disease prevention or therapy.

Informing prospective parents about their susceptibility to genetic diseases is crucial. Pre-implantation genetic diagnosis also holds significance for disease prevention by inheritance, as whole genome amplification and analysis help select a healthy embryo for implantation, preventing the transmission of a fatal metabolic disorder in the family.

Overall, the technology requires improvements in effectiveness, precision, and applications. Immunogenicity, off-target effects, mutations, delivery systems, and ethical issues are the main challenges that CRISPR technology faces. The safety concerns, ethical considerations, and the potential for misuse underscore the need for careful and responsible exploration of these technologies. CRISPR-Cas9 technology offers so much on disease prevention and treatment yet its future aspects, especially those that affect the next generations, should be investigated strictly.

Disease Treatment

CRISPR/Cas9 technology is a revolutionizing tool that can be used to combat diseases from simple to complex interventions. CRISPR technology creates targeted double-strand breaks in the human genome and provides a quick way to address genetic disorders. The primary application was targeted gene knockouts, involving the disruption of coding sequences to silence deleterious proteins. Many diseases are complex and cannot be effectively treated by simple coding sequence-targeting strategies. These diseases require more sophisticated approaches. Despite recent progress, further research is needed to develop maximally safe and effective CRISPR therapies. Although CRISPR/Cas9 technology is not actively used today, there are ongoing clinical trials focused on disease treatment. Some examples include sickle cell disease, human papillomavirus (HPV)-related cervical cancer, COVID-19 respiratory infection, renal cell carcinoma, and multiple myeloma, which are currently under clinical trial.