| Popis: |
Usher syndrome is a rare genetic disorder characterized by hearing loss and progressive vision loss, often leading to total blindness, affecting approximately 400,000 patients worldwide and making it a leading cause of deaf-blindness. While traditional treatments have focused on symptom management and support, recent advancements in gene therapy have sparked hope for potential cures. Gene therapy has shown remarkable success in treating certain retinal disorders, leading to optimism within the scientific community. However, the development and implementation of gene therapy for Usher syndrome present significant challenges. This scoping review aims to address the lack of comprehensive research into the limited translation of pre-clinical findings to clinical applications in Usher syndrome gene therapy. By identifying key obstacles at different stages of research, including pre-clinical studies, clinical trials, and economic and regulatory aspects, the review seeks to provide insights for future studies and improve the chances of developing effective gene therapy for Usher syndrome. The ultimate goal is to enhance the quality of life for individuals affected by this condition and pave the way for transformative therapeutic interventions. |
