CRISPR gene therapy: a review
TL;DR
Clinical applicability is limited by issues such off-target effects, PAM sequence restrictions, DNA damage-induced toxicity, and immunological responses to Cas proteins, despite its wide therapeutic potential, but improvements in delivery methods and high-fidelity Cas9 variations are being addressed.
Abstract
The adaptive immune system of prokaryotes is the source of CRISPR-Cas9, a ground-breaking genome editing technique that uses RNA-guided nucleases to precisely alter DNA sequences. An summary of CRISPR/Cas9's discovery, structural elements, mode of action, therapeutic uses, and present limitations is given in this article. The Cas9 nuclease and guide RNA work together to identify particular DNA targets and cause double-strand breaks in the system. Cellular processes like homology-directed repair or non-homologous end joining fix these defects, allowing for gene disruption or correction. Numerous genetic abnormalities, such as hemoglobinopathies including sickle cell disease and β-thalassemia, hereditary retinal diseases, muscular dystrophies, liver metabolic disorders, congenital lung diseases, and genetic deafness, have showed great potential for treatment with CRISPR/Cas9. Clinical applicability is limited by issues such off-target effects, PAM sequence restrictions, DNA damage-induced toxicity, and immunological responses to Cas proteins, despite its wide therapeutic potential. These obstacles are being addressed by improvements in delivery methods and high-fidelity Cas9 variations. All things considered, CRISPR/Cas9 is a revolutionary development in molecular medicine and gene therapy, providing strong prospects for accurate genome engineering and upcoming clinical uses in personalized medicine.
