Recruiting for a Gene Therapy Trial

A National Institutes of Health researcher is investigating gene therapy and hearing loss.

By Wade Chien, M.D. 

I grew up playing the violin and attended a performing arts high school in Massachusetts. I became fascinated about the ear’s ability to process complicated sounds like music and speech. It is amazing that our ears and brain can work together to instantaneously make sense out of these complex sounds. This fascination led me to become an otolaryngology (ear, nose, and throat) surgeon-scientist.

In my work I've witnessed the isolation that patients with hearing loss can experience. My patients often feel detached from their surroundings and loved ones. I feel privileged to play a role in reconnecting them through technologies like hearing aids and cochlear implants. Though these interventions have greatly improved since their invention, they remain imperfect. 

Since 2011, I have led the Inner Ear Gene Therapy Program at the National Institute on Deafness and Other Communication Disorders. Our lab focuses on developing gene therapy as a treatment for hearing loss and dizziness. We aim to restore hearing and balance by delivering normal copies of genetic materials (gene replacement) or directly modulating the genome using gene editing tools like CRISPR in animal models of hearing loss and dizziness. We have had success in improving the auditory and vestibular function in several mouse models.

Actively Recruiting Volunteers

Right now we are actively recruiting volunteers with hearing loss to participate in an outpatient research study to understand the genes that cause nonsyndromic autosomal dominant hearing loss (DFNA).

Here are the details:

  • Volunteers are healthy persons ages 3 to 99 with hearing loss that runs in the family.

  • Volunteers will undergo hearing and balance tests, a physical exam, a review of their medical history, a blood draw and genetic testing, and—for adults only—two skin biopsies.

  • Study visits may each take between two and eight hours and the study may last for several years.

  • There is no cost to participate in this study. Testing can be done at your local physician’s office or at the National Institutes of Health in Bethesda, Maryland.

My goal as a surgeon-scientist is to translate our discoveries into new therapies that will reconnect patients to the sounds and people they love. I feel immensely grateful to play a part in transforming outcomes for those affected by hearing and balance conditions.

Wade Chien, M.D., FACS, is the principal investigator leading the Inner Ear Gene Therapy Program at the National Institute on Deafness and Other Communication Disorders. He is also a professor in the department of otolaryngology–head and neck Ssrgery at the Johns Hopkins School of Medicine, with a clinical practice in Bethesda, Maryland. For information about the clinical trial, contact him at 301.443.2504 or email wade.chien@nih.gov, or visit Study Details | Natural History of Autosomal Dominant Hearing Loss | ClinicalTrials.gov.

 


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