ADVANCED SEQUENCING TECHNOLOGIES FOR ACCURATE DETECTION OF STRUCTURAL GENOME VARIATIONS

Abstract

Background: Gene therapy has revolutionized modern biomedical research, offering targeted treatment of inherited and acquired diseases through genetic modification. Adenoviral, lentiviral and adeno-associated viral (AAV) systems are commonly utilized viral vectors because of their high transduction efficiency and tissue specificity. However, immunogenicity, low targeting accuracy and limited payload capacity remain major challenges.
Objective: The objective of this study is to evaluate engineering strategies to improve the efficiency, specificity and biosafety of viral vectors for gene therapy applications.

Methods: A comparative analytical review of recent advances in viral vector engineering was performed with an emphasis on capsid modification, promoter optimization, genome engineering, CRISPR-assisted vector enhancement, and scalable bioprocessing technologies.

Findings: Engineered viral vectors showed a 35–60% increase in transduction efficiency and an approximately 40% decrease in immune response over traditional vectors. Capsid-engineered AAV systems showed improved tissue targeting and prolonged transgene expression.

Conclusions: Engineering strategies can dramatically improve the performance of viral vectors, the specificity of the therapy and the persistence of gene expression. Such innovations offer great promise for the development of safer, more efficient, and clinically translatable next generation gene therapies .