Gene therapy for hearing loss –
Source – https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10265000/
Source 2 – Click Here
Introduction
Hearing loss, a widespread health issue affecting millions globally has long lacked a curative treatment. However, recent advancements in gene therapy offer hope for those with hereditary hearing impairments.
Hereditary hearing loss, which accounts for the majority of congenital and adult-onset deafness cases, has been linked to over 130 genes. Despite the challenges, including the absence of an effective treatment, gene therapy has emerged as a potential game-changer.
The cochlea, a part of the inner ear responsible for hearing, is uniquely suited for gene therapy due to its isolation and protective barriers. Direct delivery of therapeutic vectors into the cochlear fluid space allows for targeted treatment while minimizing systemic side effects. A primary gene therapy approach involves replacing faulty genes with normal copies, a method that has shown promise in pre-clinical studies with mice models of human deafness.
Notably, recent successes in treating recessive deafness DFNB9, caused by mutations in the Otof gene, have paved the way for clinical trials. In late 2022, three clinical trials were approved to test AAV-based gene therapy for DFNB9 by companies like Sensorion, Decibel Therapeutics, and Akouos.
Gene Therapy
Despite these breakthroughs, challenges remain. Some genes essential for early cochlear development have proven resistant to AAV-based gene replacement, limiting treatment efficacy. Additionally, differences in developmental timelines between mice and humans complicate the translation of findings from mouse studies to human treatments.
Addressing these challenges requires innovative approaches, such as early in utero gene delivery, to enhance treatment efficacy. Moreover, the identification of AAV variants with high affinity for hair cells and optimization of delivery methods are crucial. Safety concerns, including potential side effects and immune responses to AAVs, also need thorough investigation.
conclusion
In conclusion, while gene therapy offers a promising avenue for treating hereditary hearing loss, further research and clinical trials are essential to ensure its safety, efficacy, and broader applicability. As scientists continue to push the boundaries of medical science, the dream of a curative treatment for hearing loss moves closer to reality.
If you need to know more please contact us – https://hearsphere.org/contact/
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