RESUMO
This article aimed to review current literature on the safety and efficacy of stem cell therapy in Stargardt disease. A comprehensive literature search was performed, and two animal and eleven human clinical trials were retrieved. These studies utilized different kinds of stem cells, including human or mouse embryonic stem cells, mesenchymal stem cells, bone marrow mononuclear fraction, and autologous bone marrow-derived stem cells. In addition, different injection techniques including subretinal, intravitreal, and suprachoroidal space injections have been evaluated. Although stem cell therapy holds promise in improving visual function in patients with Stargardt disease, further investigation is needed to determine the long-term benefits, safety, and efficacy in determining the best delivery method and selecting the most appropriate stem cell type.
Assuntos
Doença de Stargardt , Células-Tronco , Literatura de Revisão como Assunto , Distrofia Macular Viteliforme , Degeneração MacularRESUMO
La maladie de Stargardt est la plus connue des dystrophies maculaires juvéniles. Encore appelé fundus flavimaculatus, elle est une dystrophie maculaire héréditaire qui se transmet souvent sur un mode autosomique récessif et rarement sur un mode autosomique dominant. Elle est typiquement caractérisée par une déficience de la vision centrale, qui se manifeste autour de la première et ou de la deuxième décennie de la vie. Nous présentons les cas de deux frères et une soeur atteints de la maladie. La présentation, le diagnostic et les modalités thérapeutiques sont discutés.
Stargardt's disease is the best known of juvenile macular dystrophies. Also called fundus flavimaculatus, it is an inherited macular dystrophy that is often transmitted in an autosomal recessive fashion and rarely in an autosomal dominant mode. It is typically characterized by a deficiency of central vision, which manifests itself around the first and / or second decade of life. We present the cases of two brothers and one sister with the disease. Presentation, diagnosis and therapeutic modalities are discussed.