Treatment for hereditary hearing loss - wait over?
- rizaav08
- Jan 25, 2024
- 1 min read
Childrens Hospital Philadelphia have come up their initial results of experimental GENE THERAPY in treatment for a patient with hereditary hearing loss. Thanks to John. A. Germiller ,MD, PhD attending surgeon and Director of clinical research in and team at CHOP who had promising results on 11 year old child born with otoferlin gene (OTOF) mediated profound hearing loss . They where able to successfully inject small dose of gene therapy (AK-OTOF) containing copies of the normal OTOF gene directly to inner ear through round window bringing back hearing level from profound hearing loss to mild to moderate hearing loss as reflected from audiogram, first of its kind in USA.
With normal OTOF genes , the sensory cells will be able to function so they can respond to sound and activate auditory nerve to send impulses to brain. CHOP is one of several clinical trial sites in the world participating in the AK-OTOF 101 clinical trails sponsored by Akouos Inc. "As more patients at different ages are treated with this gene therapy , researchers will learn more about the degree to which hearing is improved and whether that level of hearing can be sustained over many years " Germiller said. "What we learn from following this patients progress will help direct our efforts towards helping as many patients as we can"
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