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Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions

19 Dec 2024

Abstract excerpt

Ocular gene therapy has rapidly advanced from proof-of-concept studies to clinical trials by exploiting the unique advantages of the eye, including its easy accessibility, relative immune privilege, and the ability to use the contralateral eye as a control. An important step forward was achieved with the Food and Drug Administration (FDA) approval of voretigene neparvovec (Luxturna) for the treatment of biallelic...

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PMID: 39703610
DOI: 10.59249/hwid7537

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