DefinePK

Abstract

Inherited kidney diseases, such as polycystic kidney disease (PKD), Alport syndrome, and Fabry disease, represent a significant burden on global healthcare systems due to their chronic nature and limited treatment options. Traditional therapies focus on symptom management, but they do not address the underlying genetic causes. The advent of CRISPR-Cas9 gene editing technology offers a promising avenue for correcting genetic mutations responsible for these conditions. This literature review aims to evaluate current evidence on CRISPR-based gene editing approaches for inherited kidney diseases, exploring their efficacy, safety, and challenges in preclinical and clinical settings.
A comprehensive literature search was conducted using PubMed and Google Scholar, focusing on studies from 2015 to 2025. Key search terms included "CRISPR" AND "inherited kidney disease," "gene editing" AND "polycystic kidney disease," "CRISPR" AND "Alport syndrome," and "CRISPR" AND "Fabry disease." The review includes preclinical studies, clinical trials, and systematic reviews, with a focus on CRISPR applications in kidney disease models.
Findings suggest that CRISPR-Cas9 can effectively correct mutations in genes such as PKD1, COL4A5, and GLA in cellular and animal models, leading to improved renal function and reduced disease progression. However, challenges such as off-target effects, delivery inefficiencies, and ethical concerns remain. While preclinical results are promising, clinical translation is limited, with no large-scale trials yet reported. CRISPR-based therapies may benefit specific patient populations with well-characterized mutations, but further research is needed to optimize delivery and ensure safety.
Categories: Nephrology, Genetics, Gene Therapy