Inherited Retinal Diseases

Research trends in AAV-based ocular gene therapy

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A new comprehensive study demonstrates the significant advancements and global collaboration in the field of adeno-associated virus (AAV)-based ocular gene therapy. The transition from basic research to clinical trials, coupled with the expansion of its application to diverse ocular diseases, highlights the potential of AAV-based gene therapy to revolutionize the treatment landscape for ocular conditions, including inherited retinal diseases.

As ongoing research continues to address the challenges and refine the safety and efficacy of this therapeutic approach, the future holds promising possibilities for patients seeking effective treatments for ocular diseases.

The study analyzed 832 publications related to AAV-based ocular gene therapy were from the Web of Science Core Collection, encompassing the period from 1996 to 2022.

The analysis of references and keywords revealed that the main focus areas for future research in AAV-based ocular gene therapy are “efficacy” and “safety.” This emphasis on clinical outcomes and safety underscores the transition of this research from theoretical investigations to real-world applications. Moreover, the study also highlighted the growing interest in expanding the scope of AAV-based gene therapy beyond inherited retinal diseases to include a wide range of ocular disorders.

In addition to publications, the study analyzed 80 clinical trials examining AAV-based ocular gene therapy, further substantiating the progress made in translating research findings into practical treatments. Most of these trials were conducted by institutions from the United States and Europe, underscoring their leadership and involvement in clinical research in this domain.

Chen X, Yu Y, Nie H, et al. Insights into adeno-associated virus-based ocular gene therapy: A bibliometric and visual analysis. Medicine (Baltimore). 2023;102(24):e34043. doi: 10.1097/MD.0000000000034043. PMID: 37327269; PMCID: PMC10270495.