Novel Approaches in Gene Therapy: Harnessing CRISPR/Cas9 Technology for Precision Medicine

Abstract

Gene therapy holds immense promise for the treatment of genetic disorders and other diseases by directly targeting the underlying genetic causes. This abstract provides an overview of novel approaches in gene therapy, focusing on the revolutionary CRISPR/Cas9 technology and its applications in precision medicine.The advent of CRISPR/Cas9 has revolutionized the field of gene editing, offering unprecedented precision, efficiency, and versatility in genome engineering. CRISPR/Cas9 utilizes a guide RNA (gRNA) to target specific DNA sequences, directing the Cas9 enzyme to induce double-strand breaks (DSBs) at precise genomic loci. These DSBs can be repaired through error-prone non-homologous end joining (NHEJ) or precise homology-directed repair (HDR), enabling gene knockout, gene correction, or gene insertion with unparalleled accuracy.In recent years, CRISPR/Cas9 technology has been harnessed for a wide range of applications in precision medicine, including the treatment of monogenic disorders, cancer immunotherapy, infectious disease eradication, and agricultural biotechnology. In monogenic diseases such as cystic fibrosis, sickle cell anemia, and Duchenne muscular dystrophy, CRISPR/Cas9 offers the potential to correct disease-causing mutations and restore normal gene function, paving the way for personalized gene therapies tailored to each patient's genetic makeup.Moreover, CRISPR/Cas9-mediated genome editing holds promise for cancer therapy by targeting oncogenes, tumor suppressor genes, and immune checkpoint genes implicated in tumorigenesis and immune evasion. The use of CRISPR/Cas9 to engineer T cells for chimeric antigen receptor (CAR) therapy has shown encouraging results in clinical trials, with the potential to revolutionize cancer treatment by enhancing the specificity and efficacy of immunotherapy.