脊髓小脑性共济失调7型视网膜变性:当前知识综述。

IF 1.1 4区 医学 Q3 OPHTHALMOLOGY
Arquivos brasileiros de oftalmologia Pub Date : 2025-02-24 eCollection Date: 2025-01-01 DOI:10.5935/0004-2749.2024-0248
Bruna Ferraço Marianelli, Flávio Moura Rezende Filho, Mariana Vallim Salles, José Luiz Pedroso, Orlando Graziani P Barsottini, Juliana Maria Ferraz Sallum
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引用次数: 0

摘要

脊髓小脑性共济失调7型是脊髓小脑性共济失调的一种形式,是一种临床和遗传异质性的罕见遗传性神经退行性疾病。在脊髓小脑共济失调中,小脑共济失调与锥杆视网膜营养不良之间的关联是脊髓小脑共济失调7型的有力指标。脊髓小脑共济失调7型锥杆营养不良症是一种进行性、致残且无法治愈的遗传性视网膜病变。然而,在过去的几十年里,遗传学领域取得了显著的进展,这导致了对脊髓小脑性共济失调7型视网膜变性的多个方面的理解的提高,以及其他类型视网膜营养不良的遗传治疗新模式的出现。本研究旨在评估脊髓小脑性共济失调7型视网膜变性的遗传学和分子机制,以及它们在发病机制、临床表现和潜在治疗策略方面的意义。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Retinal degeneration in spinocerebellar ataxia type 7: an overview of the current knowledge.

Spinocerebellar ataxia type 7 is a form of spinocerebellar ataxia, which is a clinically and genetically heterogeneous group of rare inherited neurodegenerative disorders. Among the spinocerebellar ataxias, the association between cerebellar ataxia and cone-rod retinal dystrophy is a strong indicator of spinocerebellar ataxia type 7. Spinocerebellar ataxia type 7 cone-rod dystrophy is a progressive, disabling, and incurable form of hereditary retinopathy. However, the field of genetics has markedly progressed in the last decades, which resulted in improved understanding of multiple aspects of spinocerebellar ataxia type 7 retinal degeneration and the emergence of new modalities of genetic therapies for other types of retinal dystrophies. This study aimed to evaluate the current knowledge on spinocerebellar ataxia type 7 retinal degeneration, including genetics and molecular mechanisms as well as their implications in pathogenesis, clinical manifestations, and potential therapeutic strategies.

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来源期刊
CiteScore
1.60
自引率
0.00%
发文量
200
审稿时长
6-12 weeks
期刊介绍: The ABO-ARQUIVOS BRASILEIROS DE OFTALMOLOGIA (ABO, ISSN 0004-2749 - print and ISSN 1678-2925 - (ABO, ISSN 0004-2749 - print and ISSN 1678-2925 - electronic version), the official bimonthly publication of the Brazilian Council of Ophthalmology (CBO), aims to disseminate scientific studies in Ophthalmology, Visual Science and Health public, by promoting research, improvement and updating of professionals related to the field.
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