The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...

Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible ...

Explore groundbreaking gene-editing therapy for kidney disease, offering hope to those with polycystic kidney disease.

- ABO-101 was well tolerated with no serious adverse events or dose-limiting toxicities reported in the 28-days following dosing - Phase 1/2 redePHine study (NCT06839235) dose escalation ongoing with ...

Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...

A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...

For decades, high cholesterol has meant a lifetime of daily pills, repeat blood tests and constant reminders that the biggest ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.