New Gene Therapy Shows Promise for Treating Hearing Loss and Balance Disorders
Researchers at the Faculty of Medical Sciences and Health at the University of Aviv have announced a breakthrough in gene therapy with the potential to treat hearing loss and balance disorders stemming from inner ear dysfunction. Published on the cover of EMBO Molecular Medicine, the study details a novel approach that successfully prevented cell degeneration in animal models, preserving both auditory and vestibular function.
Hearing loss is the most prevalent sensory disorder globally, with over half of congenital cases linked to genetic factors. The team,led by Prof. Karen Abraham and doctoral candidate Roni Hahn, alongside collaborators from Boston Hospital and Harvard Faculty (Prof. Jeffrey holt and Dr. Gwenaëlle Géléoc),aimed to develop a more effective gene therapy solution for these conditions.
Gene therapy, already demonstrating success in treating diseases like spinal muscular atrophy and congenital amaurosis Leber, utilizes modified viral vectors to deliver functional genes into cells. While adeno-associated viruses (AAVs) are commonly employed, the Aviv University team developed an advanced, structurally optimized AAV vector - dubbed AAV Vector (SCAav) – that substantially enhances gene delivery.
The study focused on a mutation in the click5 gene,crucial for the stability and function of sensory cells containing cilia within the inner ear. Deficiency in this gene leads to progressive degeneration of these cells,initially causing hearing loss and eventually impacting balance.The SCAav vector demonstrated faster and more efficient gene transmission to these sensory cells, requiring lower doses compared to customary vectors.
Remarkably, in treated animal models, the therapy halted the degeneration of ciliated cells, effectively maintaining both hearing and balance capabilities. Researchers beleive this improved efficiency offers a complete solution for patients experiencing both auditory and vestibular impairments.
“This new approach has led to an increase in therapeutic efficiency and an integrated solution for patients with both hearing loss and balance disorders,” stated the authors. They are optimistic that this research will pave the way for gene therapies addressing a broad spectrum of hereditary hearing disorders.
The project received funding from the US-Israel Science Foundation, the National Institutes of Health, and the Israel Science Foundation Breakthrough Research Programme, highlighting the collaborative nature of this significant scientific advancement.
