Gene therapy for kidney disease: targeting cystinuria.
Gene therapy for kidney disease: targeting cystinuria.
复制标题
肾脏疾病的基因治疗：靶向囊肿。
DOI：
10.1097/mnh.0000000000000768
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发表时间：
2022-03-01
影响因子：
3.2
通讯作者：
Wilson MH
中科院分区：
文献类型：
--
作者：
Peek JL;Wilson MH
本研究的目的是总结肾脏基因治疗的最新发现，同时提出胱氨酸尿症作为基因组工程治疗的模型肾脏疾病靶点。尽管基因疗法在治疗其他器官疾病方面取得了进展，但肾脏却落后了。肾脏靶向基因传递仍然是肾脏疾病基因治疗的一个障碍。纳米颗粒和腺相关病毒载体技术为肾脏基因治疗提供了新的希望。胱氨酸尿症由于其已知的遗传和分子基础、可靶向性和表型拯救能力，代表了肾脏基因治疗的一个模型潜在靶点。尽管肾脏疾病的基因治疗仍然是一个重大挑战，但新的和不断发展的技术可能实现胱氨酸尿症和其他肾脏疾病的治疗。
The aim of this study was to summarize recent findings in kidney gene therapy while proposing cystinuria as a model kidney disease target for genome engineering therapeutics. Despite the advances of gene therapy for treating diseases of other organs, the kidney lags behind. Kidney-targeted gene delivery remains an obstacle to gene therapy of kidney disease. Nanoparticle and adeno-associated viral vector technologies offer emerging hope for kidney gene therapy. Cystinuria represents a model potential target for kidney gene therapy due to its known genetic and molecular basis, targetability, and capacity for phenotypic rescue. Although gene therapy for kidney disease remains a major challenge, new and evolving technologies may actualize treatment for cystinuria and other kidney diseases.
DOI：
10.1002/dvg.23357
发表时间：
2020-05
期刊：
Genesis (New York, N.Y. : 2000)
影响因子：
--
作者：
Beckermann TM;Welch RC;Williams FM;Mortlock DP;Sha F;Ikizler TA;Woodard LE;Wilson MH
通讯作者：
Wilson MH