Screened AAV variants permit efficient transduction access to supporting cells and hair cells

Genetic malady is a significant pathogenesis underlying sensorineural hearing loss. More than half of the individuals with pre-lingual hearing loss are verified with inherited genetic defects, which are becoming a severe public health issue around the world 1 . Despite the impressive progress in identification of genes associated with deafness, no medical treatment is available for genetic hearing loss except for cochlear implantation. Therefore, developing new treatments that target diverse types of genetic hearing loss is a high priority. Inner ear gene therapy is a promising therapeutic approach to develop treatments for genetic hearing loss. The mammalian cochlea contains two types of hair cells (HCs): inner HCs (IHCs) and outer HCs (OHCs), both of which are essential for the detection and processing of auditory signal 2 . These HCs are surrounded by supporting cells (SCs), a heterogeneous group of cells that are important for cochlear homeostasis 3 . Moreover, SCs are closely associated with genetic defects, resulting in the most common hearing impairments 4 , 5 . However, the inefficiencies of gene introduction into HCs and SCs not only limit the study of the function of inner ear genes, but also hinder gene therapy application for hereditary deafness. A proper vector for efficient gene delivery into targeted HCs and SCs is a prerequisite for accurate gene therapy.