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Therapy: Gene Therapy
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Total 92 results found since Jan 2013.
Unleashing the potential of CRISPR multiplexing: Harnessing Cas12 and Cas13 for precise gene modulation in eye diseases
Vision Res. 2023 Sep 16;213:108317. doi: 10.1016/j.visres.2023.108317. Online ahead of print.ABSTRACTGene therapy is a flourishing field with the potential to revolutionize the treatment of genetic diseases. The emergence of CRISPR-Cas9 has significantly advanced targeted and efficient genome editing. Although CRISPR-Cas9 has demonstrated promising potential applications in various genetic disorders, it faces limitations in simultaneously targeting multiple genes. Novel CRISPR systems, such as Cas12 and Cas13, have been developed to overcome these challenges, enabling multiplexing and providing unique advantages. Cas13, in...
Source: Vision Research - September 18, 2023 Category: Opthalmology Authors: Fabio Bigini Soo Hyeon Lee Young Joo Sun Yang Sun Vinit B Mahajan Source Type: research
The potential for mitochondrial therapeutics in the treatment of primary open-angle glaucoma: a review
Glaucoma, an age-related neurodegenerative disease, is characterized by the death of retinal ganglion cells (RGCs) and the corresponding loss of visual fields. This disease is the leading cause of irreversible blindness worldwide, making early diagnosis and effective treatment paramount. The pathophysiology of primary open-angle glaucoma (POAG), the most common form of the disease, remains poorly understood. Current available treatments, which target elevated intraocular pressure (IOP), are not effective at slowing disease progression in approximately 30% of patients. There is a great need to identify and study treatment o...
Source: Frontiers in Physiology - August 2, 2023 Category: Physiology Source Type: research
Retinoprotective compounds, current efficacy, and future prospective
Neural Regen Res. 2023 Dec;18(12):2619-2622. doi: 10.4103/1673-5374.373662.ABSTRACTRetinal dysfunction is the most common cause of vision loss in several retinal disorders. It has been estimated a great increase in these pathologies that are becoming more globally widespread and numerous over time, also supported by the life expectancy increment. Among different types of retinopathies, we can account some that share causes, symptoms, and treatment including diabetic retinopathy, age-related macular degeneration, glaucoma, and retinitis pigmentosa. Molecular changes, environmental factors, and genetic predisposition might b...
Source: Cell Research - July 14, 2023 Category: Cytology Authors: Rachele Marino Rebecca Sappington Marco Feligioni Source Type: research
Matrix metalloproteinase-3 (MMP-3)-mediated gene therapy for glaucoma
Sci Adv. 2023 Apr 21;9(16):eadf6537. doi: 10.1126/sciadv.adf6537. Epub 2023 Apr 19.ABSTRACTApproximately 80 million people globally are affected by glaucoma, with a projected increase to over 110 million by 2040. Substantial issues surrounding patient compliance remain with topical eye drops, and up to 10% of patients become treatment resistant, putting them at risk of permanent vision loss. The major risk factor for glaucoma is elevated intraocular pressure, which is regulated by the balance between the secretion of aqueous humor and the resistance to its flow across the conventional outflow pathway. Here, we show that ad...
Source: Adv Data - April 21, 2023 Category: Epidemiology Authors: Jeffrey O'Callaghan Conor Delaney Merissa O'Connor Joseph van Batenburg-Sherwood Martin Schicht Elke L ütjen-Drecoll Natalie Hudson Sorcha Ni Dhubhghaill Peter Humphries Chris Stanley Annahita Keravala Thomas Chalberg Matthew S Lawrence Matthew Campbell Source Type: research
Gene therapy strategies for glaucoma from IOP reduction to retinal neuroprotection: progress towards non-viral systems
Adv Drug Deliv Rev. 2023 Mar 18:114781. doi: 10.1016/j.addr.2023.114781. Online ahead of print.ABSTRACTGlaucoma is the result of the gradual death of retinal ganglion cells (RGCs) whose axons form the optic nerve. Elevated intraocular pressure (IOP) is a major risk factors thatcontributes to RGC apoptosis and axonal loss at the lamina cribrosa, resulting in progressive reduction and eventual anterograde-retrograde transport blockade of neurotrophic factors. Current glaucoma management mainly focuses on pharmacological or surgical lowering of IOP, to manage the only modifiable risk factor. Although IOP reduction delays dise...
Source: Advanced Drug Delivery Reviews - March 20, 2023 Category: Drugs & Pharmacology Authors: Antoine Hakim Benjamin Guido Lokesh Narsineni Ding-Wen Chen Marianna Foldvari Source Type: research
Cell-Specific Expression of Human SIRT1 by Gene Therapy Reduces Retinal Ganglion Cell Loss Induced by Elevated Intraocular Pressure
AbstractSIRT1 prevents retinal ganglion cell (RGC) loss in several acute and subacute optic neuropathy models following pharmacologic activation or genetic overexpression. We hypothesized that adeno-associated virus (AAV)-mediated overexpression of SIRT1 in RGCs in a chronic ocular hypertension model can reduce RGC loss, thereby preserving visual function by sustained therapeutic effect. A control vector AAV-eGFP and therapeutic vector AAV-SIRT1 were constructed and optimized for transduction efficiency. A magnetic microbead mouse model of ocular hypertension was optimized to induce a time-dependent and chronic loss of vis...
Source: Neurotherapeutics - March 20, 2023 Category: Neurology Source Type: research
Adeno-associated virus mediated gene therapy for neuroprotection of retinal ganglion cells in glaucoma
Vision Res. 2023 Feb 20;206:108196. doi: 10.1016/j.visres.2023.108196. Online ahead of print.ABSTRACTGlaucoma is a group of diseases typically characterized by the degeneration of the optic nerve and is one of the world's leading causes of blindness. Although there is no cure for glaucoma, reducing intraocular pressure is an approved treatment to delay optic nerve degeneration and retinal ganglion cell (RGC) death in most patients. Recent clinical trials have evaluated the safety and efficacy of gene therapy vectors for the treatment of inherited retinal degenerations (IRDs), and the results are promising, generating enthu...
Source: Vision Research - February 22, 2023 Category: Opthalmology Authors: Christopher Starr Bo Chen Source Type: research
