Navegando por Palavras-chave "Vetores genéticos"

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    Terapia gênica em distrofias hereditárias de retina
    (Conselho Brasileiro de Oftalmologia, 2009-08-01) Côco, Monique [UNIFESP]; Han, Sang Won [UNIFESP]; Sallum, Juliana Maria Ferraz; Centro Interdisciplinar de Terapia Gênica; Universidade Federal de São Paulo (UNIFESP)
    The inherited retinal dystrophies comprise a large number of disorders characterized by a slow and progressive retinal degeneration. They are the result of mutations in genes that express in either the photoreceptor cells or the retinal pigment epithelium. The mode of inheritance can be autosomal dominant, autosomal recessive, X linked recessive, digenic or mitochondrial DNA inherited. At the moment, there is no treatment for these conditions and the patients can expect a progressive loss of vision. Accurate genetic counseling and support for rehabilitation are indicated. Research into the molecular and genetic basis of disease is continually expanding and improving the prospects for rational treatments. In this way, gene therapy, defined as the introduction of exogenous genetic material into human cells for therapeutic purposes, may ultimately offer the greatest treatment for the inherited retinal dystrophies. The eye is an attractive target for gene therapy because of its accessibility, immune privilege and translucent media. A number of retinal diseases affecting the eye have known gene defects. Besides, there is a well characterized animal model for many of these conditions. Proposals for clinical trials of gene therapy for inherited retinal degenerations owing to defects in the gene RPE65, have recently received ethical approval and the obtained preliminary results brought large prospects in the improvement on patient's quality of life.
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    Terapia gênica para osteoporose
    (Sociedade Brasileira de Ortopedia e Traumatologia, 2011-01-01) Costa, Rafael Pacheco da [UNIFESP]; Han, Sang Won [UNIFESP]; Pochini, Alberto de Castro [UNIFESP]; Reginato, Rejane Daniele [UNIFESP]; Universidade Federal de São Paulo (UNIFESP)
    Osteoporosis is considered one of the most common and serious problems affecting the elderly population worldwide. It is a chronic and progressive disease, characterized by decreased bone mass and degeneration of the microarchitecture of the bone tissue. Gene therapy represents a new approach in osteoporosis treatment, and its main function is to restore the compromised function in the metabolism. This review aims to elucidate the main studies on gene therapy in recent years, in the medical databases, that use gene therapy for the treatment of osteoporosis in animal models, as well as the future prospects of this therapy. The majority of the studies use the BMP, PTH and OPG genes, in an attempt to reestablish bone mass. Despite the lack of new molecules, all genes employed in these studies have proven to be efficient in the treatment of the disease. The benefits that gene therapy will provide for patients in the future should contribute substantially to increasing the quality of life for the elderly. Soon, clinical trials involving humans will benefit individuals with osteoporosis.