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Gene therapy and transplantation in CNS repair: the visual system
Alan R Harvey1, Ying Hu, Simone G Leaver
1School of Anatomy and Human Biology, The University of Western Australia, Crawley, WA 6009, Australia.
Abstract:
Normal visual function in humans is compromised by a range of inherited and acquired degenerative conditions, many of which affect photoreceptors and/or retinal pigment epithelium. As a consequence the majority of experimental gene- and cell-based therapies are aimed at rescuing or replacing these cells. We provide a brief overview of these studies, but the major focus of this review is on the inner retina, in particular how gene therapy and transplantation can improve the viability and regenerative capacity of retinal ganglion cells (RGCs). Such studies are relevant to the development of new treatments for ocular conditions that cause RGC loss or dysfunction, for example glaucoma, diabetes, ischaemia, and various inflammatory and neurodegenerative diseases. However, RGCs and associated central visual pathways also serve as an excellent experimental model of the adult central nervous system (CNS) in which it is possible to study the molecular and cellular mechanisms associated with neuroprotection and axonal regeneration after neurotrauma. In this review we present the current state of knowledge pertaining to RGC responses to injury, neurotrophic and gene therapy strategies aimed at promoting RGC survival, and how best to promote the regeneration of RGC axons after optic nerve or optic tract injury. We also describe transplantation methods being used in attempts to replace lost RGCs or encourage the regrowth of RGC axons back into visual centres in the brain via peripheral nerve bridges. Cooperative approaches including novel combinations of transplantation, gene therapy and pharmacotherapy are discussed. Finally, we consider a number of caveats and future directions, such as problems associated with compensatory sprouting and the reformation of visuotopic maps, the need to develop efficient, regulatable viral vectors, and the need to develop different but sequential strategies that target the cell body and/or the growth cone at appropriate times during the repair process.
Insights
Gene and cell therapies show promise for treating retinal ganglion cell (RGC) loss, a key factor in vision loss from diseases like glaucoma. Research focuses on enhancing RGC survival and axon regeneration for new vision restoration treatments.
Area of Science:
- Neuroscience
- Ophthalmology
- Regenerative Medicine
Background:
- Degenerative conditions impacting photoreceptors and retinal pigment epithelium compromise vision.
- Current therapies often target these outer retinal cells, but inner retinal strategies are crucial for conditions causing retinal ganglion cell (RGC) loss.
Purpose of the Study:
- To review gene therapy and transplantation strategies for improving retinal ganglion cell (RGC) survival and regeneration.
- To explore RGCs as a model for central nervous system (CNS) repair, focusing on neuroprotection and axonal regeneration.
Main Methods:
- Review of existing literature on gene therapy and cell transplantation for RGCs.
- Analysis of strategies for promoting RGC survival and axonal regeneration after injury.
- Discussion of combined therapeutic approaches and future research directions.
Main Results:
- Gene therapy and transplantation offer potential for rescuing or replacing RGCs.
- Understanding RGC responses to injury informs neuroprotection and regeneration strategies.
- Combined approaches may enhance therapeutic outcomes for RGC-related vision loss.
Conclusions:
- Targeting RGCs is vital for treating conditions like glaucoma, diabetes, and neurodegenerative diseases.
- Further research is needed on efficient viral vectors and sequential repair strategies.
- Developing methods for reforming visuotopic maps and managing compensatory sprouting is essential.
