Page 13 - Biotechnology newsletter 2023-24

The current trial had an OTOF mutation called DFNB9, which affects 2
to 8 percent of all cases of genetic deafness from birth. To insert the
OTOF gene into the DNA of target cells, researchers divided the gene
into two, encapsulating the halves into separate viruses, and then
injecting a mixture with both halves of the gene into the cochlea.
Although the virus injected the gene halves at different spots on the
cell’s DNA, cellular machinery assembled the complete protein,
restoring the cell’s ability to transmit signal to brain. Five of the six
children showed improvement in hearing over the 26-week trial, with
four outcomes. With hearing a critical factor in language acquisition,
researchers also measured speech perception (the ability to recognize
sound as speech) and all five of those who responded to treatment
showed improvement there.
Co-senior author of this project, Zheng-Yi Chen, who is HMS associate
professor of otolaryngology head and neck surgery and a researcher at
Mass Eye and Ear’s Eaton-Peabody Laboratories said “This really opens
the door to developing other treatments for different kinds of genetic
deafness. I’ve been working in this field for three decades, and I know
how difficult it has been to come to this point. We are at the juncture of
great future.”

~ Radhika Jhadav
T.Y.B.Sc.Biotechnology

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