Referred to the House Committee on Energy and Commerce.

Original title: “HR 9169: To support polycystic kidney disease research, and for other purposes.”

patient with aortic dilation and polycystic kidney disease

Original title: “A patient with aortic dilation and polycystic kidney disease.”

case of autosomal dominant polycystic kidney disease with multiple bilateral spontaneous retroperitoneal hemorrhages

Original title: “A case of autosomal dominant polycystic kidney disease with multiple bilateral spontaneous retroperitoneal hemorrhages.”

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other complications. This study marks the first in vivo evidence that base editing can be effective in kidney tissue, paving the way for similar approaches for other inherited kidney diseases. The research aligns with Mayo Clinic's Genesis initiative, focusing on preventing organ failure and restoring function through regenerative medicine and advanced therapies. Current treatments only slow disease progression without addressing the genetic root. Mayo Clinic's new approach uses CRISPR-based gene editing to precisely correct a DNA mutation in the PKD1 gene. Researchers developed two versions of a base editor, one for multiple organs and another specifically for kidney cells. This promising advancement directly targets the genetic mutation responsible for the condition, potentially altering the course of the disease. A single treatment of the gene therapy has shown to slow kidney cyst growth, improve heart and liver health, and extend survival in preclinical models. ROCHESTER, Minn. — Mayo Clinic researchers have made a significant breakthrough in treating autosomal dominant polycystic kidney disease (ADPKD) with a new gene-editing therapy.

Original title: “Mayo Clinic's gene therapy breakthrough targets most-common kidney disease | News | kimt.com”