Autosomal Recessive Non-Syndromic Deafness: Is AAV Gene Therapy a Real Chance?

ABSTRACT: The etiology of sensorineural hearing loss is heavily influenced by genetic mutations, with approximately 80% of cases attributed to genetic causes and only 20% to environmental factors. Over 100 non-syndromic deafness genes have been identified in humans thus far. In non-syndromic sensorineural hearing impairment, around 75-85% of cases follow an autosomal recessive inheritance pattern. In recent years, groundbreaking advancements in molecular gene therapy for inner-ear disorders have shown promising results. Experimental studies have demonstrated improvements in hearing following a single local injection of adeno-associated virus-derived vectors carrying an additional normal gene or using ribozymes to modify the genome. These pioneering approaches have opened new possibilities for potent

SUBMITTER: Brotto D 

PROVIDER: S-EPMC10961695 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature