Abstract
Vision and hearing disorders comprise the most common sensory disorders found in people. Many forms of vision and hearing loss are inherited and current treatments only provide patients with temporary or partial relief. As a result, developing genetic therapies for any of the several hundred known causative genes underlying inherited retinal and cochlear disorders has been of great interest. Recent exciting advances in gene therapy have shown promise for the clinical treatment of inherited retinal diseases, and while clinical gene therapies for cochlear disease are not yet available, research in the last several years has resulted in significant advancement in preclinical development for gene delivery to the cochlea. Furthermore, the development of somatic targeted genome editing using CRISPR/Cas9 has brought new possibilities for the treatment of dominant or gain-of-function disease. Here we discuss the current state of gene therapy for inherited diseases of the retina and cochlea with an eye toward areas that still need additional development.
Highlights
Over 2.2 billion people worldwide experience vision impairment or irreversible vision loss1, and over 466 million people worldwide experience serious hearing loss2, making these the most prevalent sensory impairments
Sound waves hit the tympanic membrane which vibrates the attached ossicular chain. This vibration is transmitted to the fluid in the cochlea and basilar membrane (Figure 1D), and resulting fluid waves are sensed by the hair cells in the organ of Corti through mechanotransduction
Another benefit of genetic therapies for both the retina and cochlea is the need to deliver only small amounts of therapeutic agents, which is due to the ability to deposit material directly to or in very close proximity to the target cells, allowing for greater chance of uptake by the desired cells with limited loss due to systemic distribution
Summary
Reviewed by: Elvir Becirovic, Ludwig Maximilian University of Munich, Germany Erica Lucy Fletcher, The University of Melbourne, Australia. Many forms of vision and hearing loss are inherited and current treatments only provide patients with temporary or partial relief. As a result, developing genetic therapies for any of the several hundred known causative genes underlying inherited retinal and cochlear disorders has been of great interest. Recent exciting advances in gene therapy have shown promise for the clinical treatment of inherited retinal diseases, and while clinical gene therapies for cochlear disease are not yet available, research in the last several years has resulted in significant advancement in preclinical development for gene delivery to the cochlea. The development of somatic targeted genome editing using CRISPR/Cas has brought new possibilities for the treatment of dominant or gain-of-function disease. We discuss the current state of gene therapy for inherited diseases of the retina and cochlea with an eye toward areas that still need additional development
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