Researchers from Purespring Therapeutics Ltd. and affiliated organizations have presented preclinical data for the adeno-associated vector (AAV) gene therapy PS-001 for the treatment of glomerular disease.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure characterized by development of multiple fluid-filled cysts linked to increased cAMP levels and fibrosis in the kidneys leading to progressive renal failure and need for dialysis or renal transplant.
Podocytes are a terminally differentiated cell type located in the glomerulus. Podocyte damage and the subsequent dysregulation of podocyte proteins have been implicated in various kidney disorders. Since gene delivery to podocytes using adeno associated vectors (AAVs) has been challenging due to various technological and physiological hurdles, investigators at Purespring Therapeutics Ltd. developed an AAV gene therapy platform that allowed for effective, specific and safe delivery of transgenes to podocytes.
Researchers from Yale School of Medicine, Ionis Pharmaceuticals Inc. and the National Institutes of Health have published data from a study that aimed to identify the signaling pathways involved in cilia-dependent cyst activation (CDCA) in autosomal dominant polycystic kidney disease (ADPKD).
Autoantibodies can promote inflammation and activate complement pathways and immune cell responses causing tissue damage in autoantibody-driven diseases. Researchers from Seismic Therapeutic Inc. have presented data on S-1117.
The generation of pathogenic autoantibodies is a crucial event in the development of inflammation and complement activation, leading to immune cell responses.
Steroid-resistant nephrotic syndrome (SRNS) is a disease characterized by hypoalbuminemia, proteinuria, edema and hyperlipidemia, and a cause of chronic kidney disease in the pediatric population.
Dysfunction of the complement system plays an important role in the pathogenesis of renal diseases. Complement inhibitors, such as C5 inhibitors, have shown efficacy in clinical trials, but may not be sufficient to block disease progression as monotherapy.
Unknown etiology is commonly encountered in the kidney pre-transplant routine program. A screening program was performed to detect patients and study recipients that meet the following features: hypertension with no clear etiology and biopsies that do not match with clinical features of classical glomerulopathies.
Nephrotic syndrome is a kidney disorder characterized by abnormal functioning of the glomerular filtration barrier, and is frequently caused by focal segmental glomerulosclerosis (FSGS). At the World Congress of Nephrology meeting this week, researchers presented a case report of a 32-year-old female patient with nephrotic syndrome caused by FSGS diagnosed when she was 27.
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