MedicalResearch.com Interview with:
Lukas Landegger MD
Molecular Neurotology Laboratory (PI Konstantina Stankovic)
Massachusetts Eye and Ear Infirmary
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: Genetic hearing loss affects more than 125 million people worldwide and constitutes a major hurdle for language acquisition and child development in general. Technological advances over the last decades, such as cochlear implants, have made it possible for deaf children to partially regain their sense of hearing. However, these devices still have several shortcomings, especially when listeners attempt to understand speech in noise or listen to music.
In establishing Anc80L65 as a reliable vector for gene delivery in the inner ear and releasing the first data demonstrating convincing hearing and vestibular function rescue in mice, we provide a foundation for other researchers interested in assessing the benefits of gene therapy in animal models of human disease.
MedicalResearch.com: What should readers take away from your report?
Response: In our study, we demonstrate for the first time that basically all important cell types in the inner ear can be reached by the new virus, including outer hair cells, which have been one of the most challenging targets to reach with currently available methods. As seen in the second paper (Pan et al. Nat Biotechnol. 2017), the ability to reach these cells carries major implications for improving hearing performance in a mouse model of human deafness, namely Usher syndrome. After the correct gene was delivered to Usher syndrome mice with the help of the novel virus, hearing improved in an unprecedented way. This finding is very promising for potential human gene therapy trials, ideally leading to the restoration of the natural ability to hear.
MedicalResearch.com: What recommendations do you have for future research as a result of this study?
Response: Additional dosing studies are in progress. We are also evaluating the importance of age at the time of injection, and the durability of the effect. If subsequent studies confirm that the therapy is safe, in accordance with the present data, we anticipate swift translation to human patients who suffer from hearing impairment.
MedicalResearch.com: Is there anything else you would like to add?
Response: We would like to thank all the funding agencies and foundations that support our research.
MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.
A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear.
Landegger LD, Pan B, Askew C, Wassmer SJ, Gluck SD, Galvin A, Taylor R, Forge A, Stankovic KM, Holt JR, Vandenberghe LH.
Nat Biotechnol. 2017 Mar;35(3):280-284. doi: 10.1038/nbt.3781. Epub 2017 Feb 6.
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