治疗罕见遗传性肾病的靶向基因疗法。

IF 14.8 1区 医学 Q1 UROLOGY & NEPHROLOGY
Veenita Khare, Stephanie Cherqui
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引用次数: 0

摘要

慢性肾脏病(CKD)是导致全球死亡的主要原因之一,其原因是肾功能衰竭以及包括透析和肾移植在内的相关治疗难题。大约三分之一的 CKD 病例与单基因遗传因素有关,因此适合进行潜在的基因治疗干预。然而,肾脏错综复杂的解剖结构带来了挑战,限制了肾脏系统靶向基因递送的有效性。在这篇综述中,我们探讨了靶向基因治疗方法领域取得的进展及其对罕见遗传性肾脏疾病的影响,研究了临床前研究和临床应用前景。体内基因治疗最常用于肾脏靶向基因递送,包括通过全身、肾静脉和肾动脉注射等不同途径给药病毒和非病毒载体。小核酸也被用于治疗某些肾脏疾病的临床前和临床研究。令人意想不到的是,造血干细胞和祖细胞已被用作肾脏基因递送的体外基因治疗载体,突出了它们在肾脏内分化成巨噬细胞的能力,形成的隧道纳米管可将遗传物质和细胞器递送到邻近的肾脏细胞,甚至可穿过基底膜,靶向近端肾小管细胞。随着基因治疗技术的不断进步和我们对肾脏生物学认识的加深,遗传性肾脏疾病患者最终有希望避免肾移植。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Targeted gene therapy for rare genetic kidney diseases.

Chronic kidney disease is one of the leading causes of mortality worldwide because of kidney failure and the associated challenges of its treatment including dialysis and kidney transplantation. About one-third of chronic kidney disease cases are linked to inherited monogenic factors, making them suitable for potential gene therapy interventions. However, the intricate anatomical structure of the kidney poses a challenge, limiting the effectiveness of targeted gene delivery to the renal system. In this review, we explore the progress made in the field of targeted gene therapy approaches and their implications for rare genetic kidney disorders, examining preclinical studies and prospects for clinical application. In vivo gene therapy is most commonly used for kidney-targeted gene delivery and involves administering viral and nonviral vectors through various routes such as systemic, renal vein, and renal arterial injections. Small nucleic acids have also been used in preclinical and clinical studies for treating certain kidney disorders. Unexpectedly, hematopoietic stem and progenitor cells have been used as an ex vivo gene therapy vehicle for kidney gene delivery, highlighting their ability to differentiate into macrophages within the kidney, forming tunneling nanotubes that can deliver genetic material and organelles to adjacent kidney cells, even across the basement membrane to target the proximal tubular cells. As gene therapy technologies continue to advance and our understanding of kidney biology deepens, there is hope for patients with genetic kidney disorders to eventually avoid kidney transplantation.

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来源期刊
Kidney international
Kidney international 医学-泌尿学与肾脏学
CiteScore
23.30
自引率
3.10%
发文量
490
审稿时长
3-6 weeks
期刊介绍: Kidney International (KI), the official journal of the International Society of Nephrology, is led by Dr. Pierre Ronco (Paris, France) and stands as one of nephrology's most cited and esteemed publications worldwide. KI provides exceptional benefits for both readers and authors, featuring highly cited original articles, focused reviews, cutting-edge imaging techniques, and lively discussions on controversial topics. The journal is dedicated to kidney research, serving researchers, clinical investigators, and practicing nephrologists.
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