Targeted Gene Therapy: Promises and Challenges in Disease Management

Abstract

Targeted gene therapy may revolutionize disease treatment by precisely treating genetic defects. This method targets particular cells or tissues with therapeutic genes to treat a variety of genetic problems, including cancer, hereditary diseases, and viral infections. Viral, nonviral, and genome editing techniques such as CRISPR-Cas9 are used for targeted gene therapy to fix or modify disease-causing genes with minimal off-target effects. The issues of vector immunogenicity, off-target mutations, and gene delivery to target cells persist despite tremendous progress. The successful implementation of targeted gene therapy is further hindered by hereditary illness complexity and genetic background diversity. These difficulties require multidisciplinary cooperation, novel vector design, and thorough preclinical and clinical assessments. The long-term and unforeseen effects of gene editing must also be considered from an ethical viewpoint. Targeted gene therapy has considerable therapeutic promise, but more research and technological advances are needed to overcome limitations and develop safe and successful clinical treatments.