Gene Therapy Techniques and Delivery Methods (Review)

TL;DR

This review explores current gene therapy technologies and delivery methods, highlighting their potential and remaining challenges in treating genetic disorders.

Contribution

The paper provides a comprehensive overview of emerging gene editing platforms and delivery systems, emphasizing the need for standardized protocols and regulatory frameworks.

Findings

CRISPR-based and novel DNA polymerase-based editors are being used for genome editing.

Delivery systems like viral vectors and lipid nanoparticles face challenges in scalability and safety.

AI integration is proposed to improve vector design and predictive analysis.

Abstract

Gene therapy has evolved into a sophisticated field encompassing diverse precision editing platforms and advanced delivery systems capable of addressing complex genetic disorders and age-related pathologies. This comprehensive review examines the current landscape of gene therapeutic technologies, including CRISPR-based genome editing, base editing systems, prime editing platforms, and emerging DNA polymerase-based editors alongside their corresponding delivery methodologies. The review encompasses viral vectors, including tissue-specific adeno-associated virus serotypes, non-viral delivery systems such as ionizable lipid nanoparticles and virus-like particles, and innovative platforms, including exosome-based delivery and the SEND system. We examine therapeutic applications spanning nuclear genome editing, mitochondrial genome modification, RNA editing, and epigenetic modulation, demonstrating the expanding scope of gene therapy beyond traditional monogenic disorders. Critical analysis reveals that while fundamental technological capabilities have been established, significant challenges remain in manufacturing scalability, long-term safety assessment, delivery across physiological barriers, and optimization of editing efficiency in post-mitotic tissues. The integration of artificial intelligence approaches for predictive analysis and rational vector design represents a promising avenue for addressing current limitations. This review concludes that successful clinical implementation requires systematic resolution of manufacturing, safety, and delivery challenges alongside the development of standardized protocols for patient stratification and robust regulatory frameworks that accommodate rapid technological innovation while ensuring patient safety.

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