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New developments in the use of gene therapy to treat Duchenne muscular dystrophy

机译：基因治疗在治疗杜氏肌营养不良症中的新进展

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Introduction: Duchenne muscular dystrophy (DMD) is a lethal X-linked inherited disorder characterised by progressive muscle weakness, wasting and degeneration. Although the gene affected in DMD was identified over 25 years ago, there is still no effective treatment. Areas covered: Here we review some of the genetic-based strategies aimed at amelioration of the DMD phenotype. A number of Phase II/III clinical trials of antisense oligonucleotide-induced exon skipping for restoration of the open reading frame (ORF) of the DMD gene have recently been completed. The potential strategies for overcoming the hurdles that appear to prevent exon skipping becoming an effective treatment for DMD currently are discussed. Expert opinion: The applicability of exon skipping as a therapy to DMD is restricted and the development of alternative strategies that are more encompassing is needed. The rapid pre-clinical advances that are being made in the field of adeno-associated virus (AAV)-based delivery of micro-dystrophin would address this. The obstacles to be faced with gene replacement strategies would include the need for high viral titres, efficient muscle targeting and avoidance of immune response to vector and transgene. The new emerging field of gene editing could potentially provide permanent correction of the DMD gene and the feasibility of such an approach to DMD is discussed.
机译：简介：杜氏肌营养不良症（DMD）是一种致命的X连锁遗传性疾病，其特征是进行性肌肉无力，消瘦和变性。尽管25年前就已经确定了DMD中受影响的基因，但仍然没有有效的治疗方法。涵盖的领域：在这里，我们回顾一些旨在改善DMD表型的基于遗传的策略。最近已经完成了许多反义寡核苷酸诱导的外显子跳跃以恢复DMD基因的开放阅读框（ORF）的II / III期临床试验。目前正在讨论克服似乎阻止外显子跳跃成为DMD有效治疗方法的障碍的潜在策略。专家意见：外显子跳跃作为DMD疗法的适用性受到限制，需要开发更具涵盖性的替代策略。在基于腺相关病毒（AAV）的微肌营养不良蛋白的递送领域中正在取得的快速的临床前进展将解决这个问题。基因替代策略将面临的障碍包括对高病毒滴度，有效的肌肉靶向以及避免对载体和转基因的免疫反应的需求。基因编辑的新领域可能潜在地提供DMD基因的永久校正，并讨论了这种DDM方法的可行性。

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