About this Research Topic
Retinal degenerative diseases such as age-related macular degeneration (AMD), diabetic retinopathy (DR), glaucoma, and retinitis pigmentosa (RP) are an increasing problem in modern society.
Impaired vision leads to a highly incapacitating condition to maintain everyday life. Blindness alone affects 34 million people worldwide. With an increasing population and its continuous aging, blinding diseases may reach epidemic levels.
Despite the advancing knowledge on genetics and molecular mechanisms of retinal degeneration processes, there is no effective cure option to date, presenting an urgent need for developing novel treatment strategies. Although some of these treatment approaches aim to rescue remaining retinal cells and thus have to be applied at early stages of the disease (e.g. gene therapy, neuroprotection, pharmacology, etc), other therapeutic interventions target late disease stages when the majority or all retinal cells have been lost, making them particularly challenging.
The aim of this Research Topic is to gather Original Articles and Reviews based on the current studies regarding the molecular and cellular mechanisms, signaling pathways, and therapy approaches in retinal degeneration, including the following topics:
Neuroprotection; Neuroprotection is a therapeutic strategy for treatment of retinal neurodegenerative disease that is independent of the degeneration etiology. It has been shown in a variety of experimental models over the last decades, that neurotrophic factors (e.g. NGF, BDNF, CNTF, GDNF), antioxidants (e.g. resveratrol, lutein, zeaxanthin, β-carotene), HDAC inhibitors, and some vitamins (e.g. vitamins A, C, and E) have the potential to delay neuronal apoptosis.
Gene therapy; An abnormal gene causing a certain disease is replaced by a normal gene in gene therapy. Adeno-associated viruses (AAV) and lentiviruses are used as tools for gene insertion in vivo and ex vivo. Recombinant AAV vectors are currently among the most frequent vectors for use in clinical trials for ocular disorders, e.g. Leber congenital amaurosis (LCA) linked to RPE65 deficiency and Choroideremia. The variability of the success rate of gene replacement therapy may be explained by the interaction of multiple factors such as the type of AAV (e.g. serotype, single- or double-stranded vector), the type of target cell, the animal model, the rate of degeneration, the offset time of treatment, the species specificity of the therapeutic transgene as well the structure, localization and molecular function of the gene product
Stem cells; The cell transplantation technique is a highly attractive strategy to replace degenerated retinal cells with healthy cells without genetic defects. Lately, retinal cells derived from induced-pluripotent stem (iPS) cells, are generating a huge impact in the field.
Retinal prostheses: Electronic prostheses are high-tech devices for electrical stimulation of internal retinal neurons and can be used to transduce light, which is the function of photoreceptor cells, and transmit it into ganglion cells in the inner retinal layer. Preclinical and clinical trials in this area continue to be studied.
Optogenetics ; The studies focused on a biological technique to induce light sensitivity in retinal cells such as bipolar cells, ganglion cells or cone photoreceptors by optogenetic approaches to target gene distribution.
Impaired vision leads to a highly incapacitating condition to maintain everyday life. Blindness alone affects 34 million people worldwide. With an increasing population and its continuous aging, blinding diseases may reach epidemic levels.
Despite the advancing knowledge on genetics and molecular mechanisms of retinal degeneration processes, there is no effective cure option to date, presenting an urgent need for developing novel treatment strategies. Although some of these treatment approaches aim to rescue remaining retinal cells and thus have to be applied at early stages of the disease (e.g. gene therapy, neuroprotection, pharmacology, etc), other therapeutic interventions target late disease stages when the majority or all retinal cells have been lost, making them particularly challenging.
The aim of this Research Topic is to gather Original Articles and Reviews based on the current studies regarding the molecular and cellular mechanisms, signaling pathways, and therapy approaches in retinal degeneration, including the following topics:
Neuroprotection; Neuroprotection is a therapeutic strategy for treatment of retinal neurodegenerative disease that is independent of the degeneration etiology. It has been shown in a variety of experimental models over the last decades, that neurotrophic factors (e.g. NGF, BDNF, CNTF, GDNF), antioxidants (e.g. resveratrol, lutein, zeaxanthin, β-carotene), HDAC inhibitors, and some vitamins (e.g. vitamins A, C, and E) have the potential to delay neuronal apoptosis.
Gene therapy; An abnormal gene causing a certain disease is replaced by a normal gene in gene therapy. Adeno-associated viruses (AAV) and lentiviruses are used as tools for gene insertion in vivo and ex vivo. Recombinant AAV vectors are currently among the most frequent vectors for use in clinical trials for ocular disorders, e.g. Leber congenital amaurosis (LCA) linked to RPE65 deficiency and Choroideremia. The variability of the success rate of gene replacement therapy may be explained by the interaction of multiple factors such as the type of AAV (e.g. serotype, single- or double-stranded vector), the type of target cell, the animal model, the rate of degeneration, the offset time of treatment, the species specificity of the therapeutic transgene as well the structure, localization and molecular function of the gene product
Stem cells; The cell transplantation technique is a highly attractive strategy to replace degenerated retinal cells with healthy cells without genetic defects. Lately, retinal cells derived from induced-pluripotent stem (iPS) cells, are generating a huge impact in the field.
Retinal prostheses: Electronic prostheses are high-tech devices for electrical stimulation of internal retinal neurons and can be used to transduce light, which is the function of photoreceptor cells, and transmit it into ganglion cells in the inner retinal layer. Preclinical and clinical trials in this area continue to be studied.
Optogenetics ; The studies focused on a biological technique to induce light sensitivity in retinal cells such as bipolar cells, ganglion cells or cone photoreceptors by optogenetic approaches to target gene distribution.
Keywords: Retina, Retinal disease, Therapy approaches, Neurodegeneration, Gene therapy
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