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Updated: May 24, 2026
Trans-Tympanic Drug Delivery for the Treatment of Ototoxicity
Published on: March 16, 2018
Gene therapy for cisplatin-induced ototoxicity: a systematic review of in vitro and experimental animal studies
Sofia Waissbluth1, Jacob Pitaro, Sam J Daniel
1McGill Auditory Sciences Laboratory, McGill University, Montreal, Quebec, Canada.
Objective:
Ototoxicity is a frequent adverse event of cisplatin treatment. No therapy is currently available for cisplatin-induced ototoxicity. A systematic review of experimental animal studies and in vitro experiments was conducted to evaluate gene therapy as a potential future therapeutic option.
Data Sources:
Eligible studies were identified through searches of electronic databases Ovid MEDLINE, Ovid MEDLINE In-Process, Embase, PubMed, Biosis Previews, Scopus, ISI Web of Science, and The Cochrane Library.
Study Selection:
Articles obtained from the search were independently reviewed by 2 authors using specific criteria to identify experimental animal studies and in vitro experiments conducted to evaluate gene therapy for cisplatin-induced ototoxicity. No restriction was applied to publication dates or languages.
Data Extraction:
Data extracted included experiment type, cell type, species, targeted gene, gene expression, method, administration, inner ear site evaluated, outcome measures for cytotoxicity, and significant results.
Results:
Fourteen articles were included in this review. In vitro and in vivo experiments have been performed to evaluate the potential of gene expression manipulation for cisplatin-induced ototoxicity. Twelve different genes were targeted including NTF3, GDNF, HO-1, XIAP, Trpv1, BCL2, Otos, Nfe2l2, Nox1, Nox3, Nox4, and Ctr1. All of the included articles demonstrated a benefit of gene therapy on cytotoxicity caused by cisplatin.
Conclusion:
Experimental animal studies and in vitro experiments have demonstrated the efficacy of gene therapy for cisplatin-induced ototoxicity. However, further investigation regarding safety, immunogenicity, and consequences of genetic manipulation in the inner ear tissues must be completed to develop future therapeutic options.
Insights
Gene therapy shows promise for preventing cisplatin-induced ototoxicity, a common side effect of cancer treatment. Further research is needed to ensure safety and efficacy before clinical application.
Area of Science:
- Oto-neuroscience
- Molecular Biology
- Gene Therapy
Background:
- Cisplatin is a widely used chemotherapy agent associated with significant ototoxicity.
- Currently, no effective treatments exist for cisplatin-induced hearing loss and tinnitus.
- Gene therapy presents a potential novel therapeutic strategy to mitigate this adverse effect.
Purpose of the Study:
- To systematically review experimental studies evaluating gene therapy for cisplatin-induced ototoxicity.
- To assess the efficacy of targeting specific genes to protect against cisplatin-induced damage.
- To identify gaps in current research for future therapeutic development.
Main Methods:
- A comprehensive systematic review of experimental animal and in vitro studies was conducted.
- Searches were performed across multiple major scientific databases without date or language restrictions.
- Data extraction focused on experimental design, targeted genes, administration methods, and outcomes related to cytotoxicity.
Main Results:
- Fourteen studies met the inclusion criteria, investigating various gene targets.
- Twelve distinct genes, including NTF3, GDNF, and HO-1, were explored for their protective effects.
- All reviewed studies reported a beneficial impact of gene therapy in reducing cisplatin-induced cytotoxicity.
Conclusions:
- Experimental evidence supports the efficacy of gene therapy in combating cisplatin ototoxicity.
- Further research is crucial to address safety, immunogenicity, and long-term effects in inner ear tissues.
- Gene therapy holds potential as a future treatment for cisplatin-induced ototoxicity, pending further investigation.
