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Gene Therapy

Gene Therapy

OVERVIEW

Gene therapy represents a transformative therapeutic paradigm that treats disease by introducing, modifying, or regulating genetic information within living cells. By directly addressing pathological mechanisms at their molecular and genetic origins, gene therapy seeks not merely to alleviate symptoms but to achieve durable, curative clinical outcomes.

The field covers a broad spectrum of modalities, including gene replacement and augmentation, genome editing, cell-based and immune cell therapies, and genetic cancer therapeutics. Realizing the full potential of gene therapy requires tightly integrated advances in disease gene discovery, mechanistic understanding of biological systems, rational therapeutic payload design, and robust delivery technologies capable of precise and sustained genetic modulation in vivo.

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In Vivo Gene Therapy and AAV Vectors

In Vivo Gene Therapy and AAV Vectors
In vivo gene therapy involves the direct delivery of therapeutic genetic payloads into patients, where clinical efficacy depends on navigating multiple biological barriers, including tissue accessibility, cellular entry, intracellular trafficking, and precise spatiotemporal control of gene expression. These challenges define the central engineering and biological bottlenecks of in vivo genetic medicine.

Among currently available delivery platforms, adeno-associated virus (AAV) vectors have emerged as the leading system for in vivo gene therapy due to their favorable safety profile, broad tissue tropism, and ability to support long-term gene expression. The clinical success of AAV-based treatments—such as Luxturna for inherited retinal diseases and Zolgensma for spinal muscular atrophy—has firmly validated the translational viability of this platform. At the same time, these successes highlight key remaining challenges, including tissue specificity, host immune responses, constrained payload capacity, and scalable manufacturing.

Our laboratory aims to address these unmet challenges through the development of next-generation AAV vectors and delivery strategies for in vivo gene therapy. By integrating vector engineering, mechanistic biology, and translational design principles, we aim to enhance targeting precision, transduction efficiency, and clinical robustness, thereby enabling broader, safer, and more effective genetic medicines.

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