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SELECT PRESENTATIONS and PUBLICATIONS

Palermo, A. (2021). Development of an AAV-Based Gene Therapy for Children With Congenital Hearing Loss Due to Otoferlin Deficiency (DB-OTO). American Society of Gene and Cell Therapy.

This presentation provided an overview of preclinical DB-OTO data from mice and non-human primates for OTOF mutation.

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Pregernig, G. (2021). Mechanisms of Promoter-Driven AAV Toxicity in the Ear. American Society of Gene and Cell Therapy.

Adeno-associated virus (AAV) is a promising approach to treat inner ear disorders. This presentation highlighted potential mechanisms of ubiquitous promoter-driven AAV toxicity in the ear.

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Viglietta, V., Shi, F., Hu, Q., Ren, Y., Keilty, J., Wolff, H., McCarthy, R., Kropp, J., Weber, P., Soglia, J. (2020). Phase 1 study to evaluate safety, tolerability and pharmacokinetics of a novel intra-tympanic administered thiosulfate to prevent cisplatin-induced hearing loss in cancer patients. Investigational New Drugs.

Ototoxicity and subsequent permanent hearing loss remain serious side effects of cisplatin, a widely used chemotherapy for solid tumors. This study reviews DB-020 efficacy and tolerability administration for cancer patients receiving cisplatin.

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Kolla, L., Kelly, M., Mann, Z., Anaya-Rocha, A., Ellis, K., Lemons, A., Palermo, A., So, K., Mays, J., Orvis, J., Burns, J., Hertzano, R., Driver, E., Kelley, M. (2020). Characterization of the development of the mouse cochlear epithelium at the single cell level. Nature communications. 

This review focuses on the analysis of collected mouse cochlear cells, including the identification of both known and unknown cell types. These cells provide insights regarding mammalian cochlear development.

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Palermo, A., Zhang, X., Arun Senapati, Goodliffe, J., Pan, B., Quigley, T., Smith, L., Weber, P., Cancelarich, S., Velez, D., Gibson, T., Becker, L., Pan, N., Sabin, L., Drummond, M., Whitton, J. (2021). Development of an AAV-Based Gene Therapy for Children With Congenital Hearing Loss Due to Otoferlin Deficiency (DB-OTO). Annual Association for Research in Otolaryngology Annual MidWinter Meeting, February 2021.

Also presented at the American Auditory Society 48th Annual Scientific and Technology Meeting, March 2021.

This presentation provided an overview of preclinical DB-OTO data from mice and non-human primates.

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Pregernig, G., So, K., Kowalczyk, M., Zhang, X., Senapati, A., Pan, B., Pan, N., Becker, L., Sabin, L., Drummond, M., Whitton, J., Palermo., A. (2021). Mechanisms of Promoter-Driven AAV Toxicity in the Ear. Annual Association for Research in Otolaryngology Annual MidWinter Meeting, February 2021.

This presentation highlighted potential mechanisms of ubiquitous promoter-driven AAV toxicity in the ear.

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Weber, P., Corrales, E., Kohler, S., Senapati, A., Goodliffe, J., Quigley, T., Becker, L., Shi, F., Gibson, T., Smith, L., Alterman, E., Enerson, B., Pan, N., Zhang, X., Drummond, M., Sabin, L., Cancelarich, S., Palermo, A., Whitton, J. (2021). Safely Delivering Adeno-Associated Viral Vectors to the Ear for Therapeutics. Annual Association for Research in Otolaryngology Annual MidWinter Meeting, February 2021.

This presentation included data in which dual vector AAV and Decibel’s cell-selective promoter drove highly selective expression of GFP in hair cells of non-human primates.

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Becker, L., Gibson, T., Koehler, S., Whitton, J., Sabin, L.,  Drummond, M., Beyman, M., Shi, F., Smith, L., Nguyen, R., Liu, S., Weber, P., Chien, W., Corrales, C., Palermo, A. (2021). Inner Ear Tropism of Natural and Engineered AAV Serotypes in Non-Human Primate Enables Therapeutic Targeting of a Diverse Set of Cochlear Cell Types. Annual Association for Research in Otolaryngology Annual MidWinter Meeting, February 2021. 

This poster demonstrated that a variety of capsids are able to transduce inner hair cells of the inner ear in non-human primates and that the presence of neutralizing antibodies was not observed to impact transduction.

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Shuohao, S., Babola, T., Pregernig, G., Bergles, D.E., Burns, J.C. & Mueller, U. (2018). Hair Cell Mechanotransduction Regulates Spontaneous Activity and Spiral Ganglion Subtype Specification in the Auditory System. Cell, 174(5), 1247-1263.

One of the major challenges to realizing the potential of synaptopathy rodent models is that current clinical audiometric approaches cannot yet reveal the presence of this subtle cochlear pathology in humans. This paper frames the key outstanding translational questions that need to be addressed to develop and realize hearing therapeutics.

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Hickox, A. E., Larsen, E., Heinz, M. G., Shinobu, L., & Whitton, J. P. (2017). Translational issues in cochlear synaptopathy. Hearing research, 349, 164-171.

Synaptopathy is undetectable by current clinical audiometric measurements in rodent models. This review outlines the key translational questions that need answering to diagnose synaptopathy.

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Burns, J. C., & Stone, J. S. (2017). Development and regeneration of vestibular hair cells in mammals. Seminars in cell & developmental biology, 65, 96-105.

Damage to the vestibular system occurs in many different cell types. This review summarizes the current state of knowledge on development, damage, and regeneration of sensory cell types in the mammalian vestibular system and highlights critical information gaps that must be addressed before new therapies for vestibular dysfunction can be defined.

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