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Purpose <p>Gene therapy is characterized as the potential for genetic enhancement by the rectification of altered genes or site-specific adjustments intended for therapeutic intervention.</p> Methods <p>It has the ability to offer in vivo and ex vivo effects that continue for an extended period. After its discovery more than half a century ago, adeno-associated virus (AAV) vectors rapidly gained attention as an excellent gene delivery vector.</p> Results <p>The growing acceptance of AAV vectors can be attributed, in particular, to their distinctive biology, simple structure, and effectiveness in gene delivery. Luxturna, which targets retinal pigment epithelial 65, was the first FDA-approved AAV-based in vivo gene therapy bioproduct. The therapy with cardiac bridging integrator 1 gene via AAV diminished the progress of heart failure, including reversing the damage by boosting heart pumping proficiency. This revolution is being prepared for human clinical trials, with FDA approval anticipated in 2025. Clinical applications employing AAV conducted for numerous diseases showed favourable consequences.</p> Conclusion <p>Nevertheless, few difficulties persist in its way, such as lower human transduction competence, inadequate methods for fabricating and purifying the virus. This therapy is expected to receive more regulatory approvals and better patient compliance with improvements in trial design, vector assembly, and novel serotypes.</p> Lay Summary <p>Gene therapy is a modern method of treating several diseases. Healthy genes are introduced into the body using a safe carrier known as the adeno-associated virus (AAV). Luxturna, the first AAV treatment authorized by the FDA, aids in the treatment of hereditary blindness. Treatments for heart failure have also shown encouraging results, and human studies are shortly to follow. Even though there are still obstacles to overcome in order to make the therapy more effective and simpler to manufacture, continued study might soon result in novel therapies for a number of dangerous illnesses.</p> Graphical Abstract <p>AAV-based gene therapy for multiple clinical applications</p> <p></p>

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Revolutionizing Gene Therapy: An Explicative Review on Engineering Adeno-associated Virus Vectors

  • 期刊论文

  • 发表日期：

    2026年2月25日

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  • Shankhadip Nandi,
  • Shaibal Chandra,
  • Kaushik Biswas,
  • Kamalika Mazumder,
  • Swarupananda Mukherjee,
  • Dipanjan Karati,
  • Soumendra Nath Bandyopadhyay,
  • Pasquale De Toro

摘要

Purpose

Gene therapy is characterized as the potential for genetic enhancement by the rectification of altered genes or site-specific adjustments intended for therapeutic intervention.

Methods

It has the ability to offer in vivo and ex vivo effects that continue for an extended period. After its discovery more than half a century ago, adeno-associated virus (AAV) vectors rapidly gained attention as an excellent gene delivery vector.

Results

The growing acceptance of AAV vectors can be attributed, in particular, to their distinctive biology, simple structure, and effectiveness in gene delivery. Luxturna, which targets retinal pigment epithelial 65, was the first FDA-approved AAV-based in vivo gene therapy bioproduct. The therapy with cardiac bridging integrator 1 gene via AAV diminished the progress of heart failure, including reversing the damage by boosting heart pumping proficiency. This revolution is being prepared for human clinical trials, with FDA approval anticipated in 2025. Clinical applications employing AAV conducted for numerous diseases showed favourable consequences.

Conclusion

Nevertheless, few difficulties persist in its way, such as lower human transduction competence, inadequate methods for fabricating and purifying the virus. This therapy is expected to receive more regulatory approvals and better patient compliance with improvements in trial design, vector assembly, and novel serotypes.

Lay Summary

Gene therapy is a modern method of treating several diseases. Healthy genes are introduced into the body using a safe carrier known as the adeno-associated virus (AAV). Luxturna, the first AAV treatment authorized by the FDA, aids in the treatment of hereditary blindness. Treatments for heart failure have also shown encouraging results, and human studies are shortly to follow. Even though there are still obstacles to overcome in order to make the therapy more effective and simpler to manufacture, continued study might soon result in novel therapies for a number of dangerous illnesses.

Graphical Abstract

AAV-based gene therapy for multiple clinical applications