Recent Advances in CRISPR-Cas9 Applications for Gene Therapy in Rare Genetic Disorders

Abstract

CRISPR-Cas9 has emerged as a groundbreaking genome-editing technology with the potential to correct genetic mutations underlying rare hereditary disorders. This study explores recent advancements in CRISPR-Cas9 applications for gene therapy, focusing on efficacy, safety, and translational potential. A systematic review of 75 peer-reviewed articles, clinical trial reports, and preclinical studies was conducted, highlighting strategies for precise gene correction, delivery mechanisms, and off-target minimization. Key findings indicate that CRISPR-mediated gene editing can restore normal gene function in conditions such as Duchenne muscular dystrophy, cystic fibrosis, and β-thalassemia. Challenges include efficient delivery to target tissues, immunogenic responses, mosaicism, and ethical concerns. The study underscores emerging approaches, including base editing, prime editing, and viral and non-viral delivery systems, that enhance specificity and clinical applicability. These advancements illustrate the transformative potential of CRISPR-Cas9 for personalized therapy in rare genetic diseases