Mayo Clinic Researchers Develop Gene Editing Therapy for Kidney Disease

A single treatment slowed kidney cyst growth, improved heart and liver health, and extended survival in preclinical models of autosomal dominant polycystic kidney disease.

Jan. 29, 2026 at 8:07pm

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Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the most common inherited kidney disorder. A single treatment of the gene therapy slowed kidney cyst growth, improved heart and liver health, and extended survival in preclinical models of ADPKD.

Why it matters

ADPKD affects an estimated 12 million people worldwide, and current treatments can only slow disease progression without addressing its genetic root. This new gene-editing approach has the potential to fundamentally change the course of the disease by correcting the underlying genetic mutation.

The details

The researchers used a form of CRISPR-based gene editing known as base editing to precisely correct a single-letter DNA mutation in the PKD1 gene. They engineered two versions of the base editor - one designed to work broadly across multiple organs and another tailored specifically to kidney cells. A single dose of the therapy corrected the PKD1 mutation in a significant proportion of cells in kidney tissue and, depending on the editor used, in the heart and liver as well.

  • The findings were published in Nature Communications on January 30, 2026.

The players

Xiaogang Li

A nephrology researcher and senior author of the study at Mayo Clinic.

Mayo Clinic

A nonprofit organization committed to clinical practice, education, and research, where the gene-editing therapy was developed.

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What they’re saying

“This is the first time we've been able to show that base editing can effectively and safely correct a disease-causing mutation in the kidney in a complex biological system.”

— Xiaogang Li, Nephrology researcher and senior author of the study

“If these approaches translate successfully to humans, they could reduce or even eliminate the need for chronic medication, delay kidney failure and significantly improve quality of life for patients with ADPKD.”

— Xiaogang Li, Nephrology researcher and senior author of the study

What’s next

Ongoing studies are focused on refining base-editing tools to address a broader range of PKD mutations, evaluating whether treatment can be effective after cysts have already formed, and developing alternative delivery methods - including nonviral options such as nanoparticles - to support future clinical use.

The takeaway

This gene-editing therapy represents a promising new approach to treating the underlying cause of autosomal dominant polycystic kidney disease, a common inherited disorder that can lead to kidney failure. If successful in human trials, it could significantly improve outcomes and quality of life for patients with this debilitating condition.