Technology ID

Transgenic Mouse Expressing Cre for the Development for Delivery of Gene Therapy

Lead Inventor
Morell, Robert (NIDCD)
Research Materials
Development Stages
Pre-Clinical (in vitro)
Research Products
Animal Models
Lead IC

This technology includes a mouse model containing a hypothetical, previously undescribed, gene that we have proven is expressed in hair cells of the inner ear and few other tissues in the body. The hair-cell limited expression of Cre is a genetic tool for creating conditional mutations affecting hair cells almost exclusively. Hair cells are the sensory receptors of both the auditory system and the vestibular system in the ears of all vertebrates.

Commercial Applications
Potentially useful for gene therapy delivery of genes to restore or protect hair cell function in the inner ear specifically.

Competitive Advantages
The ability to limit conditional mutations to just the hair cells allows for the study of hair cell development and function, without the confounding effects of pathologies in other cells in the inner ear, or other tissues in the body. Moreover, the targeted insertion does not itself cause any discernable auditory pathology in the heterozygous or homozygous state.
Licensing Contact:
Shmilovich, Michael