Nephronophthisis-Pathobiology and Molecular Pathogenesis of a Rare Kidney Genetic Disease
Nephronophthisis-Pathobiology and Molecular Pathogenesis of a Rare Kidney Genetic Disease
The exponential rise in our understanding of the aetiology and pathophysiology of genetic cystic kidney diseases can be attributed to the identification of cystogenic genes over the last three decades. The foundation of this was laid by positional cloning strategies which gradually shifted towards next-generation sequencing (NGS) based screenings. This shift has enabled the discovery of novel cystogenic genes at an accelerated pace unlike ever before and, most notably, the past decade has seen the largest increase in identification of the genes which cause nephronophthisis (NPHP). NPHP is a monogenic autosomal recessive cystic kidney disease caused by mutations in a diverse clade of over 26 identified genes and is the most common genetic cause of renal failure in children. NPHP gene types present with some common pathophysiological features alongside a diverse range of extra-renal phenotypes associated with specific syndromic presentations. This review provides a timely update on our knowledge of this disease, including epidemiology, pathophysiology, anatomical and molecular features. We delve into the diversity of the NPHP causing genes and discuss known molecular mechanisms and biochemical pathways that may have possible points of intersection with polycystic kidney disease (the most studied renal cystic pathology). We delineate the pathologies arising from extra-renal complications and co-morbidities and their impact on quality of life. Finally, we discuss the current diagnostic and therapeutic modalities available for disease management, outlining possible avenues of research to improve the prognosis for NPHP patients.
- UNSW Sydney Australia
- Macquarie University Australia
- Children's Hospital at Westmead Australia
High-Throughput Nucleotide Sequencing, Humans, Genetic Predisposition to Disease, Review, Sequence Analysis, DNA, Kidney Diseases, Cystic
High-Throughput Nucleotide Sequencing, Humans, Genetic Predisposition to Disease, Review, Sequence Analysis, DNA, Kidney Diseases, Cystic
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