Navegando por Palavras-chave "retinal degeneration"

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    Correction of Monogenic and Common Retinal Disorders with Gene Therapy
    (Mdpi, 2017) Sengillo, Jesse D.; Justus, Sally; Cabral, Thiago [UNIFESP]; Tsang, Stephen H.
    The past decade has seen major advances in gene-based therapies, many of which show promise for translation to human disease. At the forefront of research in this field is ocular disease, as the eye lends itself to gene-based interventions due to its accessibility, relatively immune-privileged status, and ability to be non-invasively monitored. A landmark study in 2001 demonstrating successful gene therapy in a large-animal model for Leber congenital amaurosis set the stage for translation of these strategies from the bench to the bedside. Multiple clinical trials have since initiated for various retinal diseases, and further improvements in gene therapy techniques have engendered optimism for alleviating inherited blinding disorders. This article provides an overview of gene-based strategies for retinal disease, current clinical trials that engage these strategies, and the latest techniques in genome engineering, which could serve as the next frontline of therapeutic interventions.
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    New stratusoct findings in extrapapillary excavated anomaly and secondary macular complications
    (Lippincott Williams & Wilkins, 2007-01-01) Aggio, Fabio Bom [UNIFESP]; Farah, Michel E.; Meirelles, Rodrigo L.; Costa, Rogerio A.; Monteiro, Mario L. R.; Universidade Federal de São Paulo (UNIFESP); Universidade de São Paulo (USP)
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    STRATUS optical coherence tomography in unilateral colobomatous excavation of the optic disc and secondary retinoschisis
    (Springer, 2005-01-01) Meirelles, R. L.; Aggio, F. B.; Costa, R. A.; Farah, M. E.; Universidade Federal de São Paulo (UNIFESP)
    Purpose: To report the STRATUS optical coherence tomography (STRATUSOCT) findings in a patient with unilateral coloboma- like excavation of the optic disc without pit but secondary retinoschisis, as well as to discuss the possible involved pathophysiologic mechanisms. Methods: Observational case report. STRATUSOCT findings in a 66-year-old woman with a coloboma-like excavation of the optic disc without pit but secondary retinoschisis encompassing the macular region, along with evidence of a mild epiretinal membrane superonasal to the disc were evaluated. Results: STRATUSOCT showed signs of a connection between the perineural space and the inner retinal layers on the temporal optic disc border, as well as schisis-like changes extending from the disc to the macula, with cystoid degeneration and two lamellar holes in their nasal portion. Conclusion: the use of third generation OCT afforded an enhanced visualization of retinal structures, revealing signs of fluid at several distinct levels, as well as deep and superficial inner breaks apart from the schisis cavity. We are unaware of such previous reports, and could find no reference to them in a computerized search using MEDLINE. in addition, our study supports a common pathomechanism for the development of macular complications in optic pits and colobomas.