OTOF Gene Therapy: From Breakthroughs to Roadmaps

References

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[3] O. Akil, F. Dyka, C. Calvet, et al., “Dual AAV-Mediated Gene Therapy Restores Hearing in a DFNB9 Mouse Model,” Proceedings of the National Academy of Sciences of the United States of America 116, no. 10 (2019): 4496–4501.

[4] J. Lv, H. Wang, X. Cheng, et al., “AAV1-hOTOF Gene Therapy for Autosomal Recessive Deafness 9: A Single-Arm Trial,” Lancet 403, no. 10441 (2024): 2317–2325.

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