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Genetic Kidney Diseases (GKDs) Modeling Using Genome Editing Technologies

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Laboratorio de Nefroloxía (No. 11), Grupo de Xenética e Bioloxía do Desenvolvemento das Enfermidades Renais, Instituto de Investigación Sanitaria de Santiago (IDIS), Complexo Hospitalario de Santiago de Compostela (CHUS), 15706 Santiago de Compostela, Spain
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Grupo de Medicina Xenómica, Centro Singular de Investigación en Medicina Molecular y Enfermedades Crónicas (CiMUS), 15706 Santiago de Compostela, Spain
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Fundación Pública Galega de Medicina Xenómica-SERGAS, Complexo Hospitalario de Santiago de Compostela (CHUS), 15706 Santiago de Compostela, Spain
*
Authors to whom correspondence should be addressed.
Academic Editor: Tetsushi Sakuma
Cells 2022, 11(9), 1571; https://doi.org/10.3390/cells11091571
Received: 31 March 2022 / Revised: 29 April 2022 / Accepted: 4 May 2022 / Published: 6 May 2022
Genetic kidney diseases (GKDs) are a group of rare diseases, affecting approximately about 60 to 80 per 100,000 individuals, for which there is currently no treatment that can cure them (in many cases). GKDs usually leads to early-onset chronic kidney disease, which results in patients having to undergo dialysis or kidney transplant. Here, we briefly describe genetic causes and phenotypic effects of six GKDs representative of different ranges of prevalence and renal involvement (ciliopathy, glomerulopathy, and tubulopathy). One of the shared characteristics of GKDs is that most of them are monogenic. This characteristic makes it possible to use site-specific nuclease systems to edit the genes that cause GKDs and generate in vitro and in vivo models that reflect the genetic abnormalities of GKDs. We describe and compare these site-specific nuclease systems (zinc finger nucleases (ZFNs), transcription activator-like effect nucleases (TALENs) and regularly clustered short palindromic repeat-associated protein (CRISPR-Cas9)) and review how these systems have allowed the generation of cellular and animal GKDs models and how they have contributed to shed light on many still unknown fields in GKDs. We also indicate the main obstacles limiting the application of these systems in a more efficient way. The information provided here will be useful to gain an accurate understanding of the technological advances in the field of genome editing for GKDs, as well as to serve as a guide for the selection of both the genome editing tool and the gene delivery method most suitable for the successful development of GKDs models. View Full-Text
Keywords: Genetic kidney diseases; ZFN; TALEN; CRISPR-Cas9; disease modeling; GKDs models Genetic kidney diseases; ZFN; TALEN; CRISPR-Cas9; disease modeling; GKDs models
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MDPI and ACS Style

Gómez-García, F.; Martínez-Pulleiro, R.; Carrera, N.; Allegue, C.; Garcia-Gonzalez, M.A. Genetic Kidney Diseases (GKDs) Modeling Using Genome Editing Technologies. Cells 2022, 11, 1571. https://doi.org/10.3390/cells11091571

AMA Style

Gómez-García F, Martínez-Pulleiro R, Carrera N, Allegue C, Garcia-Gonzalez MA. Genetic Kidney Diseases (GKDs) Modeling Using Genome Editing Technologies. Cells. 2022; 11(9):1571. https://doi.org/10.3390/cells11091571

Chicago/Turabian Style

Gómez-García, Fernando, Raquel Martínez-Pulleiro, Noa Carrera, Catarina Allegue, and Miguel A. Garcia-Gonzalez. 2022. "Genetic Kidney Diseases (GKDs) Modeling Using Genome Editing Technologies" Cells 11, no. 9: 1571. https://doi.org/10.3390/cells11091571

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