Gene Therapy Milestone

For 11 children in China it was nothing short of a miracle.  They received pioneering gene therapy during clinical trials and the results have been life changing for them, and offer hope to anyone suffering from hearing loss.

They were all born deaf due to DFNB9, an inherited condition caused by mutations in the OTOF gene.  The results of two recent studies have proven that the therapy developed by researchers at Mass Eye and Ear in Boston and Fudan University in Shanghai is both safe and effective.  For the first time in their lives the children were able to hear and gained close to normal hearing.

One of the lead researchers, Dr. Zheng-li Chen, holds out hope that their work will eventually lead to treatments for many forms of deafness and hearing loss.“This gene therapy is really to show the proof of concept of this approach,” says Chen. “It is a gateway for us to develop treatments for other types of hearing loss, whether it’s genetic, or non-genetic causes, like aging and noise-induced damage, which affect millions of people.”

You can learn about another promising research program led by Dr. Chen here. And if you would like to support Dr. Chen’s work, consider making a donation at Donate Mass Eye & Ear

In this video, courtesy of Mass Eye and Ear, Dr. Chen explains the groundbreaking clinical trials.

 

Author: Digby Cook

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