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Inspired by rare genetic disease, researchers develop a compound that can protect kidney cells from death and restore kidney function in multiple animal models of progressive kidney disease.

Ó³»­´«Ã½ scientists deploy RNA sequencing to nail down disease-causing gene mutations in patients for whom genetic analysis failed to return a diagnosis. The study demonstrates the power of RNA sequencing to augment standard diagnostic tools in the clinic.

Researchers, clinicians, patient advocates, and industry representatives gathered recently at the Ó³»­´«Ã½ for the third annual Glom-NExT Symposium (Glom-NExT3), organized by institute member of the Ó³»­´«Ã½, Anna Greka. With the goal of bringing stakeholders together to discuss advancing therapeutics for kidney disease, the symposium featured talks and a roundtable discussion with patient advocates, FDA representation and thought leaders in the field. Due to popular demand, the meeting has been rebranded as Kidney-NExT and will be coming back to Ó³»­´«Ã½ next spring.

An unusual case of a rare anemia is opening scientists up to a new way of thinking about how to adapt and employ cytokines (messenger molecules of the blood and immune system) as tools for treatment — a way that is more analog than digital — and illustrating the promise of precision medicine.

The study of structural variation — large-scale changes in DNA that can, in some cases, refashion entire chromosomes — in the genomic era has lagged behind that of sequence variation. But there’s a growing appreciation of how important structural variants are to human biology and disease. What makes these variants more challenging to study, and what is being done to overcome those challenges?

Ó³»­´«Ã½ researchers Sonia Vallabh and Eric Minikel joined President Obama, Vice President Biden, and select members of Congress for the signing of legislation that provides for investments in research and development, and delivery of treatments for disease.