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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
OBJECTIVE To determine the safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) carrying the RPE65 gene.Expand
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A high-density integrated genetic linkage and radiation hybrid map of the laboratory rat.
The laboratory rat (Rattus norvegicus) is a key animal model for biomedical research. However, the genetic infrastructure required for connecting phenotype and genotype in the rat is currentlyExpand
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Carbohydrate feedings 1 h before exercise improves cycling performance.
The effects of consuming two different amounts of liquid carbohydrate 1 h before exercise on the metabolic responses during exercise and on exercise performance were determined. Subjects consumedExpand
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Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.
Abstract Proof of concept for MERTK gene replacement therapy has been demonstrated using different viral vectors in the Royal College of Surgeon (RCS) rat, a well characterized model of recessiveExpand
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Long-term outcomes in eyes receiving fixed-interval dosing of anti-vascular endothelial growth factor agents for wet age-related macular degeneration.
PURPOSE To report on long-term visual outcomes in patients receiving continuous fixed-interval dosing of anti-vascular endothelial growth factor (VEGF) treatment in neovascular age-related macularExpand
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Ab-Externo AAV-Mediated Gene Delivery to the Suprachoroidal Space Using a 250 Micron Flexible Microcatheter
Background The current method of delivering gene replacement to the posterior segment of the eye involves a three-port pars plana vitrectomy followed by injection of the agent through a 37-gaugeExpand
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DILUTE POVIDONE-IODINE PROPHYLAXIS MAINTAINS SAFETY WHILE IMPROVING PATIENT COMFORT AFTER INTRAVITREAL INJECTIONS.
PURPOSE To report the rates of postintravitreal injection (IVT) endophthalmitis with topical conjunctival application of various concentrations of povidone-iodine (PI), including no PI. METHODSExpand
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RaScaL: A Pilot Study to Assess the Efficacy, Durability, and Safety of a Single Intervention with Ranibizumab plus Peripheral Laser for Diabetic Macular Edema Associated with Peripheral Nonperfusion
Background and Objectives: To determine the efficacy, durability, and safety of a single treatment with intravitreal ranibizumab plus peripheral scatter laser (RaScaL) in patients with diabeticExpand
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Variegated yet non-random rod and cone photoreceptor disease patterns in RPGR-ORF15-associated retinal degeneration.
Mutations in the ORF15 exon of the RPGR gene cause a common form of X-linked retinitis pigmentosa, which often results in severe loss of vision. In dogs and mice, gene augmentation therapy has beenExpand
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Intravitreal bevacizumab treatment of bilateral peripapillary choroidal neovascularization from optic nerve head drusen.
Choroidal neovascular membranes are a rare cause of decreased vision in children with optic nerve head drusen. We present a case of bilateral choroidal neovascular membranes associated with opticExpand
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