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Gene therapy for pulmonary hypertension: prospects and challenges.

机译:肺动脉高压的基因治疗:前景和挑战。

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INTRODUCTION: Recent evidence shows that pulmonary arterial hypertension (PAH) remains a fatal disease despite the introduction of new pharmacological treatments. New options are therefore needed and gene therapy approaches are a rational consideration based on emerging understanding of the genetic basis of PAH. AREAS COVERED: This review briefly discusses the recent developments in clinical management of PAH and the investigation of gene delivery techniques for pulmonary vascular disease from 1997 to 2010, relating this to improved understanding of disease pathogenesis during this period. There is a focus on bone morphogenetic protein receptor type 2, as mutations in this gene are clearly linked to disease pathogenesis and outcomes. The reader will gain insight into the gene vector strategies being used, the target cells and the specific genes being delivered as candidate therapeutic approaches for PAH. EXPERT OPINION: Various genes and strategies for delivery have achieved improvements in PAH in animal models, which is encouraging for the development of this technology for human application. The main limiting factor for clinical progress relates to gene delivery vector technology.
机译:简介:最近的证据表明,尽管采用了新的药物治疗方法,肺动脉高压(PAH)仍然是一种致命疾病。因此,需要新的选择,并且基于对PAH遗传基础的新认识,基因治疗方法是一个合理的考虑因素。覆盖的领域:这篇综述简要讨论了1997年至2010年PAH临床管理的最新进展以及肺血管疾病基因传递技术的研究,这与人们对该时期疾病发病机理的了解有关。人们将重点放在2型骨形态发生蛋白受体上,因为该基因的突变与疾病的发病机制和结果密切相关。读者将深入了解正在使用的基因载体策略,靶细胞和作为PAH候选治疗方法的特定基因。专家意见:各种基因和递送策略已在动物模型中实现了PAH的改善,这为该技术在人类应用中的发展提供了令人鼓舞的信息。临床进展的主要限制因素涉及基因递送载体技术。

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