Background: Congenital hearing loss, a prevalent permanent paediatric sensory impairment affecting multiple births globally, significantly impacts communicative and cognitive development. Due to the lack of cochlea regenerative capacity, traditional management through auditory amplification fails to achieve natural restoration. Recent advancements in stem cell therapy present a promising regenerative approach, showing potential for hair cell regeneration and auditory function restoration. Objective: This review provides an analysis of the efficacy and safety of stem cell intervention for congenital hearing loss, highlighting key research advancements, challenges, and future research directions, emphasising the need for interdisciplinary approaches and innovative technologies. Methods: A literature search was conducted across databases such as Cochrane, Google Scholar, PubMed and Web of Science. Studies were included if they were published between 2015 and 2025, in English, and focused on stem cell therapy, cochlea regeneration, hearing recovery and regenerative cell therapy. Results: The review included 65 studies. Significant progress is evident using stem cell therapy for cochlea hair cell regeneration and auditory nerve function restoration. Key findings include using gene therapy for cochlea cell reprogramming and using stem cells for hair cell regeneration. While preclinical models show promising results, human clinical trials indicate moderate improvements in hearing function, with challenges of cell integration, long-term efficacy, safety concerns, tumorigenesis and immune rejection noted. Conclusion: Stem cell therapy holds promise for potentially curing congenital hearing loss through cochlea regeneration and hearing recovery. Significant challenges and the need for optimised therapeutic protocols remain. Further research should include large-scale clinical trials, improved stem cell differentiation efficiency, refined gene editing technologies, and better delivery mechanisms, which are essential to realise the full therapeutic potential of stem cell intervention.
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