CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive review
Genetic kidney diseases are caused by mutations in specific genes that significantly affect kidney development and function. Although the underlying pathogenic genes of many kidney diseases have been identified, an understanding of their mechanisms and effective treatments remains limited. Gene edit...
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| Format: | Article |
| Language: | English |
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Korean Society of Pediatric Nephrology
2025-02-01
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| Series: | Childhood Kidney Diseases |
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| Online Access: | http://chikd.org/upload/ckd-25-008.pdf |
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| author | Seungyeon Kang Se Jin Park Min Ho Lee Andreas Kronbichler Jae Il Shin |
| author_facet | Seungyeon Kang Se Jin Park Min Ho Lee Andreas Kronbichler Jae Il Shin |
| author_sort | Seungyeon Kang |
| collection | DOAJ |
| description | Genetic kidney diseases are caused by mutations in specific genes that significantly affect kidney development and function. Although the underlying pathogenic genes of many kidney diseases have been identified, an understanding of their mechanisms and effective treatments remains limited. Gene editing, particularly using clustered regularly interspaced short palindromic repeats (CRISPR), has recently become a promising approach for studying genetic diseases and the CRISPR/CRISPR-associated protein 9 (CRISPR-Cas9) method has become a prominent research method. It has been shown that CRISPR-Cas9 can be targeted to knock out specific genomic sites, which enables researchers to correct gene mutations, prevent inheritance, and better understand the function of genes and the effectiveness of drugs. However, the application of CRISPR-Cas9 technology in the development of therapeutic agents against genetic kidney disease has been overlooked compared with other genetic diseases. In this paper, we provide an overview of the current research advancements in genetic kidney diseases using CRISPR technology, as well as the diverse preclinical research methods implemented, with particular emphasis on autosomal dominant polycystic kidney disease. |
| format | Article |
| id | doaj-art-039e732bd983463fb5f200f355d4a1da |
| institution | DOAJ |
| issn | 2384-0242 2384-0250 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Korean Society of Pediatric Nephrology |
| record_format | Article |
| series | Childhood Kidney Diseases |
| spelling | doaj-art-039e732bd983463fb5f200f355d4a1da2025-08-20T03:14:47ZengKorean Society of Pediatric NephrologyChildhood Kidney Diseases2384-02422384-02502025-02-0129141110.3339/ckd.25.008824CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive reviewSeungyeon Kang0Se Jin Park1Min Ho Lee2Andreas Kronbichler3Jae Il Shin4 Department of Pharmacology and Toxicology, Molecular Genetics and Microbiology, University of Toronto, Toronto, ON, Canada Department of Pediatrics, Changwon Hanmaeum Hospital, Hanyang University College of Medicine, Changwon, Republic of Korea Department of Orthopedic Surgery, Yonsei University Health System, Seoul, Republic of Korea Department of Internal Medicine IV, Nephrology and Hypertension, Medical University of Innsbruck, Innsbruck, Austria Department of Pediatrics, Yonsei University College of Medicine, Seoul, Republic of KoreaGenetic kidney diseases are caused by mutations in specific genes that significantly affect kidney development and function. Although the underlying pathogenic genes of many kidney diseases have been identified, an understanding of their mechanisms and effective treatments remains limited. Gene editing, particularly using clustered regularly interspaced short palindromic repeats (CRISPR), has recently become a promising approach for studying genetic diseases and the CRISPR/CRISPR-associated protein 9 (CRISPR-Cas9) method has become a prominent research method. It has been shown that CRISPR-Cas9 can be targeted to knock out specific genomic sites, which enables researchers to correct gene mutations, prevent inheritance, and better understand the function of genes and the effectiveness of drugs. However, the application of CRISPR-Cas9 technology in the development of therapeutic agents against genetic kidney disease has been overlooked compared with other genetic diseases. In this paper, we provide an overview of the current research advancements in genetic kidney diseases using CRISPR technology, as well as the diverse preclinical research methods implemented, with particular emphasis on autosomal dominant polycystic kidney disease.http://chikd.org/upload/ckd-25-008.pdfautosomal dominant polycystic kidney diseaseclustered regularly interspaced short palindromic repeatscrispr-associated protein 9gene editingkidney |
| spellingShingle | Seungyeon Kang Se Jin Park Min Ho Lee Andreas Kronbichler Jae Il Shin CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive review Childhood Kidney Diseases autosomal dominant polycystic kidney disease clustered regularly interspaced short palindromic repeats crispr-associated protein 9 gene editing kidney |
| title | CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive review |
| title_full | CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive review |
| title_fullStr | CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive review |
| title_full_unstemmed | CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive review |
| title_short | CRISPR-Cas9 system in autosomal dominant polycystic kidney disease: a comprehensive review |
| title_sort | crispr cas9 system in autosomal dominant polycystic kidney disease a comprehensive review |
| topic | autosomal dominant polycystic kidney disease clustered regularly interspaced short palindromic repeats crispr-associated protein 9 gene editing kidney |
| url | http://chikd.org/upload/ckd-25-008.pdf |
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