التفاصيل البيبلوغرافية
| العنوان: |
Towards the Clinical Application of Gene Therapy for Genetic Inner Ear Diseases |
| المؤلفون: |
Ghizlene Lahlou, Charlotte Calvet, Marie Giorgi, Marie-José Lecomte, Saaid Safieddine |
| المصدر: |
Journal of Clinical Medicine; Volume 12; Issue 3; Pages: 1046 |
| بيانات النشر: |
Multidisciplinary Digital Publishing Institute |
| سنة النشر: |
2023 |
| المجموعة: |
MDPI Open Access Publishing |
| مصطلحات موضوعية: |
gene therapy, hearing impairment, vestibular defect, AAV |
| الوصف: |
Hearing loss, the most common human sensory defect worldwide, is a major public health problem. About 70% of congenital forms and 25% of adult-onset forms of deafness are of genetic origin. In total, 136 deafness genes have already been identified and there are thought to be several hundred more awaiting identification. However, there is currently no cure for sensorineural deafness. In recent years, translational research studies have shown gene therapy to be effective against inherited inner ear diseases, and the application of this technology to humans is now within reach. We provide here a comprehensive and practical overview of current advances in gene therapy for inherited deafness, with and without an associated vestibular defect. We focus on the different gene therapy approaches, considering their prospects, including the viral vector used, and the delivery route. We also discuss the clinical application of the various strategies, their strengths, weaknesses, and the challenges to be overcome. |
| نوع الوثيقة: |
text |
| وصف الملف: |
application/pdf |
| اللغة: |
English |
| العلاقة: |
Otolaryngology; https://dx.doi.org/10.3390/jcm12031046Test |
| DOI: |
10.3390/jcm12031046 |
| الإتاحة: |
https://doi.org/10.3390/jcm12031046Test |
| حقوق: |
https://creativecommons.org/licenses/by/4.0Test/ |
| رقم الانضمام: |
edsbas.BFA86F9F |
| قاعدة البيانات: |
BASE |
