[salubrioustoxin]

Yes. Also, even within rare disease this application has desirable features. Obvious bio marker (hearing), relatively isolated organ system with obvious delivery method (inject into cochlea). main downside is that there is an existing alternative (implant) so risk/benefit question is less obvious compared to eg neurodegenerative diseases.

[DoingIsLearning]

Is delivery critical or just get it close enough to tissue in ROI?

Are we aiming to modify specific cells/layers? And what happens to adjacent tissue that is 'accidentally' modified?

Do you have any literature recommendations on the state of the art in Gene Therapy 'delivery' mechanisms/apparatus?