CRISPR therapy: A revolutionary breakthrough in genetic medicine

Gene editing with CRISPR-Cas9 technology revolutionizes modern medicine by enabling precise DNA modifications. Initially discovered as a bacterial defense mechanism, the Cas9 enzyme, guided by RNA, can target and cut specific DNA sites, allowing for gene editing. Applications include genetic engineering, functional studies, and potential treatments for genetic diseases like cancer. Notably, in 2023, CRISPR-Cas9 was approved for treating sickle cell anaemia with significant results, despite challenges like long-term security and high costs. CRISPR-Cas9 technology allows for correcting genetic defects, treating diseases, and improving agricultural crops. It can regulate gene transcription through the CRISPRi system, using an inactive Cas9 to interfere with gene expression without permanently altering DNA. This gene-editing tool shows promise in gene therapy, potentially curing diseases like HIV-1, sickle cell disease, and haemophilia B. However, challenges include off-target mutations and efficient delivery of CRISPR/Cas9. Precise target site selection and dosage control are crucial, with tools like CasOT helping identify and prevent unwanted mutations. CRISPR-Cas9 requires a PAM sequence to function, narrowing its targets in the genome but increasing specificity. Production of gRNA faces challenges due to mRNA processing, with alternatives like the artificial gene RGR showing promise. Efficient delivery methods are still needed, with current techniques involving DNA and RNA injection. Future applications include treating genetic diseases and agricultural improvements, with ongoing research essential for overcoming challenges and ensuring safety and accuracy. Global collaboration is vital for the ethical use of this technology. Contribution to evidence-based healthcare: CRISPR therapy represents an exciting frontier in genetic medicine, allowing for precise gene editing and opening up new possibilities for treating incurable diseases.