Deafness Gene Therapy

December 28, 2017

Confocal microscopy images of mouse cochlea; hair cells in green. (Left) An untreated cochlea from a mouse with the Tmc1 mutation, displaying hair cell loss. (Right) The opposite, treated, cochlea from the same mouse, displaying hair cell preservation. (Gao et al. Nature 2017)

BOB HIRSHON (host):

Halting progressive deafness. I’m Bob Hirshon and this is Science Update.

Hair cells are the sound-sensing structures of our inner ear. But they can be silenced by a mutation of a single gene known as TMC1, or Beethoven.

ZHENG-YI CHEN (Harvard Medical School and Massachusetts Eye and Ear Infirmary):

Due to the presence of the mutation, the hair cell will die eventually, and as a result, the patient will not be able to hear.

HIRSHON:

That’s Harvard researcher Zheng-Yi Chen. In the journal Nature, he and his colleagues report successfully snipping out the bad gene in a mouse model and halting the disorder. They used a technology called CRISPR to design a protein complex that could be injected into the confined space around the hair cells. Chen says this localized gene repair poses fewer risks than other methods. Partnering with a team in China, they are now refining the therapy on pigs, and hope to then move to human trials. I’m Bob Hirshon, for AAAS, the science society.

Story by Bob Hirshon