Chiorini, John (Jay) (NIDCR)
Di Pasquale, Giovanni (NIDCR)
Zorzi, Veronica (CNR)
Mammano, Fabio (Universita degli Studi di Padova (UNIPD))
This technology includes a novel AAV isolate (AAV44.9) to be used as gene therapy for the inner ear for the treatment of deafness. The ability of AAV vectors to transduce dividing and non-dividing cells, establish long-term transgene expression, and the lack of pathogenicity has made them attractive for use in gene therapy applications. Vectors based on new AAV isolates may have different host range and different immunological properties, thus allowing for more efficient transduction in certain cell types. We recently identified a novel AAV isolate as a contaminant of a laboratory stock of adenovirus and is referred to as AAV44.9. Surprisingly, this isolate has high gene transfer activity in a number of cell types including salivary gland cell, liver and nerves. Hearing and balance depend on the function of the inner ear sensory epithelium, which consists of hair cells and a number of supporting cells that provide mechanical support for the sensory cells. The development of efficient transgene delivery for the inner ear is an important step toward potential application of gene-based therapies for cochlear disorders.
Gene transfer for deafness and specifically targeting hair cells.
Enhanced spread and transduction of hair cells in the inner ear.