Current trends in gene therapy for retinal diseases (Review)

Moraru, Andreea; Costin, D; Iorga, Raluca Eugenia; Munteanu, Mihnea; Moraru, Radu Lucian; Brănișteanu, Dumitru

doi:10.3892/etm.2021.10948

Cited by 11 publications

(11 citation statements)

References 35 publications

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“…Historically, some of the most prominent clinical targets for PL include amblyopia (Levi & Li, 2009), cortical visual impairments (Pollock et al, 2019;Saionz et al, 2021), refractive errors (Polat et al, 2012), low vision in youths (Nyquist et al, 2016) or older adults (DeLoss et al, 2014), and restoration of vision following removal of juvenile cataracts (Kalia et al, 2017). New challenges emerge as treatments for vision loss increasingly involve prosthetics-whether electronic versions applied to the retina (e.g., Argus II, Palanker, 2023) or cortex (cortical prostheses, Fine & Boynton, 2023), or through optogenetics (Provansal et al, 2022), gene therapies (Moraru et al, 2022), or stem cell transplantation (Öner, 2018). For instance, it is increasingly clear that new criteria and methods are needed to assess, and improve through training, the "ultra-low vision" created by these approaches (Ayton et al, 2020).…”

Section: Applications To Vision Lossmentioning

confidence: 99%

“…Pathology can originate within the visual system or be secondary to more generalized neurological or mental conditions such as multiple sclerosis, major depression, schizophrenia, dementia, and autism, or to systemic processes such as abnormalities of metabolism, genetics, immune function, and even normal aging. Historically, some of the most prominent clinical targets for PL include amblyopia (Levi & Li, 2009), cortical visual impairments (Pollock et al, 2019; Saionz et al, 2021), refractive errors (Polat et al, 2012), low vision in youths (Nyquist et al, 2016) or older adults (DeLoss et al, 2014), and restoration of vision following removal of juvenile cataracts (Kalia et al, 2017). New challenges emerge as treatments for vision loss increasingly involve prosthetics—whether electronic versions applied to the retina (e.g., Argus II, Palanker, 2023) or cortex (cortical prostheses, Fine & Boynton, 2023), or through optogenetics (Provansal et al, 2022), gene therapies (Moraru et al, 2022), or stem cell transplantation (Öner, 2018).…”

Section: Clinical Applications Of Plmentioning

confidence: 99%

See 1 more Smart Citation

Perceptual Learning: Policy Insights From Basic Research to Real-World Applications

Seitz,

Sekuler,

Dosher

et al. 2023

Policy Insights from the Behavioral and Brain Sciences

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Perceptual learning is the process by which experience alters how incoming sensory information is processed by the brain to give rise to behavior—it is critical for how humans educate children, train experts, treat diseases, and promote health and well-being throughout the lifespan. Knowledge of perceptual learning requires basic and applied research in humans and nonhuman animal models, which informs strategic targets for advancing applications. Commercial products to induce perceptual learning are proliferating rapidly with limited regulation (e.g., for rehabilitation), while at the same time basic science is increasingly restricted by changing regulations (such as new granting-agency definitions of clinical trials). Realizing the full potential of perceptual learning requires balancing basic and translational science to advance new knowledge, while serving and protecting consumers. Reforms can promote open, accessible, and representative research, and the translation of this research to applications across different sectors of society.

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Section: Applications To Vision Lossmentioning

confidence: 99%

Section: Clinical Applications Of Plmentioning

confidence: 99%

Perceptual Learning: Policy Insights From Basic Research to Real-World Applications

Seitz,

Sekuler,

Dosher

et al. 2023

Policy Insights from the Behavioral and Brain Sciences

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“…However, although these issues make POAG a difficult ocular disease to diagnose and treat, they also present several opportunities to intervene at multiple loci in the disease process to help retard the progression of the visual impairment and thus help preserve the eyesight of the affected patients. Some of the factors and other elements shown in Figure 2 are amenable to well-established methods to help discover and develop drugs, including gene therapy ( Figure 3 ) [ 7 , 13 , 14 ], as investigated in animal models of eye disorders and in some cases in human subjects with varying degrees of success [ 7 , 8 , 9 , 14 ].…”

Section: Pathogenesis Of Coht and Paogmentioning

confidence: 99%

“…Consequently, vision is critical for our survival and good quality of life since our ability to read, learn, socialize, and perform daily tasks are highly eyesight-dependent [ 1 , 2 ]. Unfortunately, as with other organs, the eye is prone to various dysfunctions and diseases such as ocular hypertension (OHT), glaucoma, age-related macular degeneration, dry eye, diabetic retinopathy and many inherited retinal diseases (IRDs) [ 1 , 2 , 3 , 4 , 5 , 6 , 7 , 8 , 9 ]. The current review aims to describe the etiology of chronic OHT (cOHT) and one specific type of glaucoma, primary open-angle glaucoma (POAG), which is mainly caused by chronically elevated intraocular pressure (IOP) or OHT [ 3 , 4 , 5 , 6 , 7 ].…”

Section: Introductionmentioning

confidence: 99%

Elevated Intraocular Pressure and Glaucomatous Optic Neuropathy: Genes to Disease Mechanisms, Therapeutic Drugs, and Gene Therapies

Sharif

2023

Pharmaceuticals

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This review article focuses on the pathogenesis of and genetic defects linked with chronic ocular hypertension (cOHT) and glaucoma. The latter ocular disease constitutes a group of ocular degenerative diseases whose hallmark features are damage to the optic nerve, apoptotic demise of retinal ganglion cells, disturbances within the brain regions involved in visual perception and considerable visual impairment that can lead to blindness. Even though a number of pharmaceuticals, surgical and device-based treatments already exist addressing cOHT associated with the most prevalent of the glaucoma types, primary open-angle glaucoma (POAG), they can be improved upon in terms of superior efficacy with reduced side-effects and with longer duration of activity. The linkage of disease pathology to certain genes via genome-wide associated studies are illuminating new approaches to finding novel treatment options for the aforementioned ocular disorders. Gene replacement, gene editing via CRISPR-Cas9, and the use of optogenetic technologies may replace traditional drug-based therapies and/or they may augment existing therapeutics for the treatment of cOHT and POAG in the future.

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“…[64][65][66] The current limitations in ocular gene therapy include the autoimmune response for the viral DNA and safe and efficient delivery of the gene therapy in proximity to the target tissue, namely the choroid, RPE and retina. [67,68] The use of non-viral nanoparticles (NPs) as a therapeutic alternative is becoming increasingly popular because of their unique properties and biocompatibility. The development of a nanocarrier that can accurately and efficiently deliver this complex into the cytosol of eye cell/tissue and facilitate the journey toward the nucleus is an exciting area of research (Figure 2).…”

Section: Red-emissive N-cds For Porcine Ocular Globe Imagingmentioning

confidence: 99%

Functional Carbon Quantum Dots for Ocular Imaging and Therapeutic Applications

Kumar

Sher

Rencus‐Lazar

et al. 2022

Small

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functional groups such as CHO, CO, NH 2 , OH, COOH, COC, CN, NO, SH, or polymer aggregates. [11] Moreover, CDs are considered to be the most recent form of zero-dimensional carbon, with a diameter of less than 10 nm and excellent fluorescent properties. [12] These properties have led to their application in biomedical engineering [13] and sensor technology, [14,15] including heavy metal ion detection, [16] fluorescent labeling and nucleus targeted imaging of cells, [17] targeting of drug delivery for anti-tumor therapy, [18] photovoltaic devices/solar cell, [19] photocatalysts, [20] dye degradation, [21] lubricant additive, [22] supercapacitor, [23] nanoelectronic devices, [24] fuel cells, [25] anti-viral therapy, [26] photodynamic therapy, [27] gene delivery, [28] and disruption of the fibrillation of amyloidogenic peptides, such as Aβ. [29,30] Recently, Shah et al. have investigated the presence of Aβ in aging eyes and its significance in relation to Alzheimer's disease (AD) pathology. [31] They have also discussed clinical studies investigating the in vivo ocular imaging of Aβ and its relationship to brain Aβ burden, as well as therapies that target ocular Aβ. [31] Therefore, the characterization of retinal Aβ is also necessary in order to differentiate it from eye-related diseases.According to the World Health Organization, more than 2.2 billion individuals were affected by some form of visual impairment in 2020, with an estimated 35 million blind individuals. [32][33][34] Among the leading causes of vision loss Carbon quantum dots (CDs) are a class of emerging carbonaceous nanomaterials that have received considerable attention due to their excellent fluorescent properties, extremely small size, ability to penetrate cells and tissues, ease of synthesis, surface modification, low cytotoxicity, and superior water dispersion. In light of these properties, CDs are extensively investigated as candidates for bioimaging probes, efficient drug carriers, and disease diagnostics. Functionalized CDs represent a promising therapeutic candidate for ocular diseases. Here, this work reviews the potential use of functionalized CDs in the diagnosis and treatment of eye-related diseases, including the treatment of macular and anterior segment diseases, as well as targeting Aβ amyloids in the retina.

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Current trends in gene therapy for retinal diseases (Review)

Cited by 11 publications

References 35 publications

Perceptual Learning: Policy Insights From Basic Research to Real-World Applications

Perceptual Learning: Policy Insights From Basic Research to Real-World Applications

Elevated Intraocular Pressure and Glaucomatous Optic Neuropathy: Genes to Disease Mechanisms, Therapeutic Drugs, and Gene Therapies

Functional Carbon Quantum Dots for Ocular Imaging and Therapeutic Applications

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