Research progress of gene therapy in polycystic kidney disease
10.3760/cma.j.cn441217-20240320-00328
- VernacularTitle:多囊肾病的基因治疗进展
- Author:
Xinming LI
1
;
Zhiguo MAO
;
Changlin MEI
;
Cheng XUE
Author Information
1. 海军军医大学第二附属医院（上海长征医院）肾内科 解放军肾脏病研究所，上海 200003
- Keywords:
Polycystic kidney diseases;
Gene therapy;
Oligonucleotides antisense;
CRISPR-Cas9;
Adeno-associated virus vector
- From:
Chinese Journal of Nephrology
2024;40(11):905-911
- CountryChina
- Language:Chinese
-
Abstract:
Polycystic kidney disease (PKD) is a hereditary kidney disease characterized by the formation of numerous cysts in the kidneys, which progressively impairs renal function over time. PKD is primarily divided into two types: autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD), with ADPKD being more prevalent. Current treatments primarily focus on symptom relief and disease progression delay, lacking a curative approach. However, the development of gene editing technologies such as clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (CRISPR-Cas9) and adeno-associated virus (AAV) vectors has offered new therapeutic possibilities for ADPKD and ARPKD. These include approaches like antisense oligonucleotides (ASO), adenovirus-mediated gene knockdown, CRISPR- Cas9, Pkd1 gene enhancement therapy, and the use of induced pluripotent stem cells (iPSCs), which have shown potential efficacy in animal models and early clinical studies. Despite facing technological challenges, ethical and legal issues, and high costs, gene therapy presents an unprecedented hope for PKD treatment. Future interdisciplinary collaboration and international cooperation are essential for developing more effective treatment strategies for PKD patients.
