AAV-mediated genetic interventions in the treatment of rare kidney disease: a narrative review

Gene therapy for rare diseases marks a new era in precision medicine: Insights from clinical trials

Clinical evidence suggests that gene-based therapies in the management of rare diseases can achieve sustained functional benefits, reduce disease-related complications, and lessen dependence on long-term replacement or supportive treatments.

Reviewing Traditional and Novel Approaches to Managing Anemia of Chronic Kidney Disease

Anemia is a common complication of chronic kidney disease (CKD). If not managed appropriately, anemia can be highly debilitating and potentially life-threatening for CKD patients. Various treatments have been developed to alleviate CKD-associated anemia, with the main goals of restoring circulating iron, promoting erythropoiesis, and downregulating hepcidin. Mainstream treatment options include iron supplements, erythropoiesis-stimulating agents (ESAs), hepcidin antagonists, and blood transfusion. However, the therapeutic efficacy of current CKD anemia treatments is hindered by extensive adverse effects, most notably iron overload and ESA resistance. HIF-PH (hypoxia-induced factor prolyl hydroxylase) inhibitors are a novel class of orally bioavailable drugs for CKD anemia. HIF-PH inhibitors adopt a distinct mechanism, stimulating erythropoiesis via transcriptional regulation of the hypoxia-activated erythropoietin (EPO) gene. This review summarizes existing and emerging therapeutic strategies for CKD-associated anemia, with particular emphasis on HIF-PH inhibitors and their clinical potential and risks.

Chronic kidney disease‑associated encephalopathy: Clinical features, pathomechanisms and emerging therapeutic strategies (Review).

Chronic kidney disease (CKD) is a progressive disorder whose systemic effects extend to the central nervous system, leading to CKD‑associated encephalopathy. Epidemiological data indicate that the prevalence of cognitive impairment in patients with CKD is as high as 40%, while mood disorders such as depression and anxiety exceed 60% among those receiving hemodialysis, and the risk of cerebrovascular events is also notably increased. These complications substantially impair the quality of life of patients, functional independence and long‑term prognosis, thereby constituting a considerable clinical burden. The key pathological mechanisms involve disruption of the 'gut‑kidney‑brain axis'. Declining renal function leads to the accumulation of gut microbiota‑derived uremic toxins, such as indoxyl sulfate, p‑Cresyl sulfate and trimethylamine N‑oxide. Through multiple pathways, including disruption of blood‑brain barrier integrity, induction of neuroinflammation, promotion of oxidative stress and direct neurotoxicity, these toxins collectively contribute to the injury of the neurovascular unit, neuronal dysfunction and even neurodegeneration. The present review systematically outlines the clinical manifestations, the aforementioned core pathogenic mechanisms and emerging therapeutic strategies for CKD‑associated encephalopathy. Clinically, early identification of neurological complications (such as through neuropsychological assessment, gait analysis and neuroimaging), along with monitoring of specific biomarkers, is crucial for timely intervention and improved prognosis. In terms of treatment, the targeted interventions on the gut‑kidney‑brain axis (such as specific probiotics and intestinal adsorbents), and the advantages and disadvantages of stem cells and gene therapy are summarized in the present review. A deeper understanding of these mechanisms will provide a solid theoretical foundation for the development of innovative treatments and ultimately improve neurological outcomes in patients with CKD.

ADVANCES IN IGA NEPHROPATHY: NOVEL THERAPEUTIC STRATEGIES AND CLINICAL OUTCOMES

Immunoglobulin A nephropathy is the most common primary glomerular disease worldwide and remains a leading cause of chronic kidney disease and end-stage kidney failure. The disease is characterized by mesangial deposition of galactose-deficient immunoglobulin A1 (Gd-IgA1)-containing immune complexes, which initiate complement activation, glomerular inflammation, and progressive renal fibrosis. Despite decades of research, conventional treatment strategies have largely focused on supportive therapy, including optimized blood pressure control, renin–angiotensin system blockade, and lifestyle modification. However, advances in the understanding of IgAN pathogenesis have led to the development of novel targeted therapies that address key pathogenic pathways. These include targeted-release budesonide, endothelin receptor antagonists, complement inhibitors, B-cell activating factor inhibitors, APRIL-targeted therapies, sodium-glucose cotransporter-2 inhibitors, and other emerging immunomodulatory agents. In parallel, advances in biomarker discovery, multi-omics technologies, artificial intelligence, and precision medicine are improving risk stratification and enabling individualized therapeutic approaches. This review summarizes current knowledge regarding the pathophysiology of IgA nephropathy, recent therapeutic innovations, clinical outcomes, and future directions for personalized disease management.

Research Progress and Critical Challenges of Bioartificial Kidneys in Renal Replacement Therapy for End-Stage Renal Disease

Chronic kidney disease (CKD), one of the major global public health burdens, continues to exhibit a rising prevalence worldwide. The growing population of patients with end-stage renal disease (ESRD) has led to an increasing demand for renal replacement therapy (RRT). Although dialysis effectively prolongs survival, it fails to fully replicate kidney function. In addition, the persistent shortage of donor kidneys results in prolonged waiting periods for kidney transplantation. Emerging renal replacement strategies, such as kidney organoids, have demonstrated considerable potential. However, multiple technical limitations continue to hinder their near-term clinical translation. Bioartificial kidneys (BAKs), which integrate engineering and biological technologies, generally consist of artificial filtration membranes and living-cell bioreactors designed to mimic native kidney function. Advances in nanotechnology, biomaterials, and tissue engineering have accelerated the development of implantable bioartificial kidneys (iBAKs), making them an important research direction in renal replacement therapy. These innovations have improved membrane performance, biocompatibility, and cellular integration; however, substantial challenges remain regarding long-term stability, immune compatibility, and clinical validation before translation into human applications. Specifically, limited cell sources and uncertain long-term biocompatibility remain major barriers to iBAK development. In the future, biosensors and artificial intelligence (AI) technologies may be incorporated into bioartificial kidneys to enable personalized precision therapy. This review focuses on the developmental and major challenges of bioartificial kidneys, with detailed discussion of recent progress in implantable artificial kidneys.

synthe-Reviewing traditional and novel approaches to Managing anemia of Chronic Kidney Disease