Cochlear Gene Therapy for Sensorineural Hearing Loss: Current Status and Major Remaining Hurdles for Translational Success
| Autor: | Yong Feng, Wen-Wen Wang, Sun Myoung Kim, Xi Erick Lin, Wen-Juan Zhang, Cuiyuan Cai, Weijia Kong |
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| Rok vydání: | 2018 |
| Předmět: |
0301 basic medicine
medicine.medical_specialty Hearing loss medicine.medical_treatment Genetic enhancement review Psychological intervention Sound perception genetic mutations Audiology sensorineural hearing loss lcsh:RC321-571 03 medical and health sciences Cellular and Molecular Neuroscience genetic deafness Cochlear implant otorhinolaryngologic diseases medicine lcsh:Neurosciences. Biological psychiatry. Neuropsychiatry Molecular Biology Cochlea preclinical trials business.industry viral-mediated gene expression medicine.disease cochlear gene therapy Clinical trial 030104 developmental biology hearing restoration Sensorineural hearing loss medicine.symptom business Neuroscience |
| Zdroj: | Frontiers in Molecular Neuroscience, Vol 11 (2018) Frontiers in Molecular Neuroscience |
| ISSN: | 1662-5099 |
| DOI: | 10.3389/fnmol.2018.00221 |
| Popis: | Sensorineural hearing loss (SNHL) affects millions of people. Genetic mutations play a large and direct role in both congenital and late-onset cases of SNHL (e.g., age-dependent hearing loss, ADHL). Although hearing aids can help moderate to severe hearing loss the only effective treatment for deaf patients is the cochlear implant (CI). Gene- and cell-based therapies potentially may preserve or restore hearing with more natural sound perception, since their theoretical frequency resolution power is much higher than that of cochlear implants. These biologically-based interventions also carry the potential to re-establish hearing without the need for implanting any prosthetic device; the convenience and lower financial burden afforded by such biologically-based interventions could potentially benefit far more SNHL patients. Recently major progress has been achieved in preclinical studies of cochlear gene therapy. This review critically evaluates recent advances in the preclinical trials of gene therapies for SNHL and the major remaining challenges for the development and eventual clinical translation of this novel therapy. The cochlea bears many similarities to the eye for translational studies of gene therapies. Experience gained in ocular gene therapy trials, many of which have advanced to clinical phase III, may provide valuable guidance in improving the chance of success for cochlear gene therapy in human trials. A discussion on potential implications of translational knowledge gleaned from large numbers of advanced clinical trials of ocular gene therapy is therefore included. |
| Databáze: | OpenAIRE |
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