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Often called a silent killer, many people may have no idea they carry a gene that would lead to a higher chance of developing kidney disease. The ability to identify genes which code for molecules that lead to kidney disease allows
In a recent Op-Ed in Stat, Dr. Maddux advocated for using genetic markers to reclassify kidney disease which he believes will help increase efforts to develop these more precise treatments.
“I want to see all kidney disease patients have a robust pipeline of innovative therapeutic alternatives and be aware of any trials referencing their specific condition,” said Dr. Maddux.
In a separate study published in the Proceedings of the National Academy of Sciences (PNAS), researchers at Newcastle University discovered a potential way to halt the progression of kidney disease in people with Joubert syndrome. For the first time, researchers used gene editing in a mouse model to trick cells into bypassing the faulty gene that causes kidney failure in this specific disease.
"We now know how we may be able to offer a therapy that corrects the gene mistake within kidney cells and prevent the development of genetic kidney disease,” said Professor John Sayer from the Institute of Genetic Medicine at Newcastle University.
While this exciting development may still be years away from an actual therapy for people with Joubert syndrome, it’s a perfect example of why these genetic markers are so critical for developing new targeted therapies using the latest gene editing techniques.
Insight: Translating Science into Practice: FMCNA is an Ecosystem that Drives Progress by Dr. Frank Maddux
Stat Op-ed: Kidney disease is a killer. More precise classification can help tame it by Dr. Frank Maddux
Insight: Personalized Nephrology by Len Usvyat