Book chapter
Gene and cell-based therapies for retinal and optic nerve disease
Cell and Gene Therapies for Neurologic Diseases, pp.243-262
Handbook of Clinical Neurology, v. 205, Elsevier Health Sciences
2024
DOI: 10.1016/B978-0-323-90120-8.00016-2
PMID: 39341657
Abstract
Leading causes of blindness worldwide include neurodegenerative diseases of the retina, which cause irreversible loss of retinal pigment epithelium (RPE) and photoreceptors, and optic neuropathies, which result in retinal ganglion cell (RGC) death. Because photoreceptor and RGCs do not spontaneously regenerate in mammals, including humans, vision loss from these conditions is, at present, permanent. Recent advances in gene and cell-based therapies have provided new hope to patients affected by these conditions. This chapter reviews the current state and future of these approaches to treating ocular neurodegenerative disease. Gene therapies for retinal degeneration and optic neuropathies primarily focus on correcting known pathogenic mutations that cause inherited conditions to halt progression. There are multiple retinal and optic neuropathy gene therapies in clinical trials, and one retinal gene therapy is approved in the United States, Canada, Europe, and Australia. Cell-based therapies are mutation agnostic and have the potential to repopulate neurons regardless of the underlying etiology of degeneration. While photoreceptor cell replacement is nearing a human clinical trial, RPE transplantation is currently in phase I/II clinical trials. RGC replacement faces numerous logistical challenges, but preclinical research has laid the foundation for functional repair of optic neuropathies to be feasible.
Details
- Title: Subtitle
- Gene and cell-based therapies for retinal and optic nerve disease
- Creators
- Edward P. Esposito - Johns Hopkins UniversityIan C. Han - University of IowaThomas V. Johnson - Johns Hopkins Medicine
- Resource Type
- Book chapter
- Publication Details
- Cell and Gene Therapies for Neurologic Diseases, pp.243-262
- Publisher
- Elsevier Health Sciences
- Series
- Handbook of Clinical Neurology; v. 205
- DOI
- 10.1016/B978-0-323-90120-8.00016-2
- PMID
- 39341657
- ISSN
- 0072-9752
- Number of pages
- 20
- Language
- English
- Date published
- 2024
- Academic Unit
- Ophthalmology and Visual Sciences
- Record Identifier
- 9984721242702771
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