The United Kingdom’s Medicines Healthcare Products Regulatory Agency (MHRA) has authorized the first-in-human clinical trial testing Sangamo Therapeutics’ TX200, a cell-based CAR-Treg therapy, in preventing rejection of transplanted kidneys in patients with end-stage renal disease (ESRD). Alport syndrome patients are at high risk of…
Pregnancy may exacerbate the impact of COL4A4 gene mutations in women who have recessive, or non-symptomatic forms of Alport syndrome, a case report shows. Published in the journal Nephron, the case report of a 31-year-old woman diagnosed postpartum with autosomal recessive Alport syndrome (ARAS) highlights the importance…
A rare type of mutation in the COL4A5 gene in a young boy led to severe X-linked Alport syndrome, according to a case report. The report, “Somatic Mosaicism in a Male Patient With X-linked Alport Syndrome,” was published in the journal Kidney International Reports. Alport syndrome is caused…
While Alport syndrome currently remains without a cure, ongoing research is testing several strategies that hold promise, a review study shows. The review, “New therapeutic options for Alport syndrome,” was published in the journal Nephrology Dialysis Transplantation. Alport syndrome is an inherited disease caused by mutations in the…
Reata Pharmaceuticals has completed patient enrollment for Phase 3 of its CARDINAL study investigating the safety and efficacy of bardoxolone methyl (bardoxolone) for improving kidney function in people with Alport syndrome, the company announced. The Phase 3 part of the CARDINAL trial (NCT03019185), which has enrolled…
Children and adolescents with Alport syndrome who underwent kidney transplants are at higher risk for developing kidney infections by BK virus, a South Korean study suggests. The study, “Higher Incidence of BK Virus Nephropathy in Pediatric Kidney Allograft Recipients with Alport Syndrome,” was published in the Journal of…
Using stem cells derived from Alport syndrome (AS) patients to examine their genetic and protein profile, scientists identified three factors — microRNA-4775, ELV-like protein 1-A (ELAVL1), and epidermal growth factor receptor (EGFR) — as likely candidates in this genetic disease’s underlying mechanisms, and possible targets for treatments. The study “…
Increased urinary excretion of the CD80 protein was found in all patients with active kidney diseases, including Alport syndrome, in a recent study, rendering the protein an unreliable diagnostic biomarker to differentiate between renal diseases, the researchers report. The study, “The utility of urinary CD80 as a diagnostic marker in…
Using induced pluripotent stem cells may help researchers identify the underlying mechanisms of Alport syndrome, according to a new study from Monash University in Austrialia. Researchers developed podocyte-like cells — cells important for the filtering capacity of the kidneys — from two Alport syndrome patients and found that the…
A small-inhibitor molecule targeting a protein called lysyl oxidase like-2 or LOXL2 was seen to lessen renal scarring and improve the workings of the kidney in a mouse model of Alport syndrome — raising the possibility it can slow disease progression. The study, “Lysyl oxidase like-2 contributes to renal…
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