Progress in Gene Editing Tools and Their Potential for Correcting Mutations Underlying Hearing and Vision Loss

Area of Science:

• Genetics
• Ophthalmology
• Otolaryngology
• Molecular Biology

Background:

• Blindness and deafness are common sensory disorders often caused by genetic, environmental, or age-related factors.
• Damage to photoreceptor cells (vision) and hair cells (hearing) can lead to irreversible sensory loss.
• Current research focuses on gene therapies to correct or replace disease-causing genetic mutations.

Purpose of the Study:

• To review the current applications and achievements of gene-based therapies for genetic sensory disorders.
• To highlight the potential of new gene editing technologies in treating blindness and deafness.
• To discuss the challenges and solutions for successful in vivo therapeutic applications in the eye and ear.

Main Methods:

• Review of gene replacement therapies for inherited retinal diseases.
• Analysis of preclinical studies using gene silencing and editing for deafness.
• Examination of CRISPR/Cas nucleases, base, prime, and RNA editors for mutation repair.
• Discussion of the eye and ear as privileged sites for genetic therapy development.

Main Results:

• Successful gene therapy product for a childhood hereditary blindness.
• Promising preclinical results for auditory function restoration in deafness models.
• Application of gene editing tools like CRISPR/Cas for various genetic forms of blindness and deafness.
• Advancements in DNA and RNA-based editors for direct mutation repair.

Conclusions:

• The eye and ear are leading areas for developing genetic therapies due to accessibility and immune privilege.
• New gene editing technologies offer significant potential for treating genetic blindness and deafness.
• Overcoming challenges is crucial for the successful in vivo therapeutic application of these emerging treatments.