UCLA Researchers Develop Nanoparticle-Based Gene Therapy for Cystic Fibrosis

New approach packages entire therapeutic gene and editing machinery into a single non-viral delivery system

Published on Feb. 20, 2026

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UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory model of cystic fibrosis. The study shows that lipid nanoparticles can carry the complex molecular cargo required for precise insertion of a large full-length gene into the genome without using viral vectors.

Why it matters

Cystic fibrosis is caused by mutations in a single gene, and about 10% of patients produce little or no functional protein, leaving them with no options for existing drug treatments. This new gene therapy approach offers a potential universal solution that could work across many different disease-causing mutations.

The details

The researchers engineered lipid nanoparticles to transport three gene-editing components simultaneously: CRISPR machinery to cut DNA at a precise location, guide molecules to target the correct genomic site, and a DNA template encoding a full, functional copy of the CFTR gene. In lab-grown human airway cells carrying a severe cystic fibrosis mutation, the nanoparticles successfully delivered the healthy CFTR gene into about 3-4% of the cells, but this small fraction of corrected cells restored 88-100% of normal CFTR channel function across the cell population.

  • The study was published in February 2026.

The players

UCLA Broad Stem Cell Research Center

A research center at the University of California, Los Angeles focused on stem cell research and regenerative medicine.

Dr. Steven Jonas

Senior author of the study and a member of the UCLA Broad Stem Cell Research Center.

Dr. Brigitte Gomperts

Co-author of the study and associate director of translational research at the UCLA Broad Stem Cell Research Center, as well as a professor of pediatrics and pulmonary medicine at the David Geffen School of Medicine at UCLA.

Ruth Foley

The study's first author and a recent Ph.D. graduate from the Jonas lab at UCLA.

Dr. Donald Kohn

Collaborator whose lab developed the codon optimization technique used to maximize protein production from the replacement CFTR gene.

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What they’re saying

“This work shows that we can package everything needed for precise gene insertion into a single, non-viral delivery system. That's a critical step toward developing gene therapies that can work across many different disease-causing mutations.”

— Dr. Steven Jonas, Senior author of the study and member of the UCLA Broad Stem Cell Research Center (Mirage News)

“For those patients, gene therapy isn't just an improvement — it's really the only option. You have to give the cell the ability to make the protein in the first place.”

— Dr. Brigitte Gomperts, Co-author of the study and associate director of translational research at the UCLA Broad Stem Cell Research Center (Mirage News)

“Getting all of that into a single particle — especially a gene as large as CFTR — is something that hadn't been shown before. If you can solve the 'big gene' problem, it opens the door for a lot of other diseases as well.”

— Ruth Foley, First author of the study and recent Ph.D. graduate from the Jonas lab at UCLA (Mirage News)

What’s next

The researchers say the next challenge is getting the gene-editing nanoparticles to reach the airway stem cells, which sit deep within the lung's protective lining and regenerate the airway throughout a person's life. Reaching those cells is crucial for achieving long-term, durable benefits from the gene therapy.

The takeaway

This nanoparticle-based gene-editing approach represents a promising new path toward mutation-agnostic gene therapy for cystic fibrosis and potentially other genetic lung diseases. By packaging the entire therapeutic gene along with the gene-editing machinery, the researchers have overcome a key challenge in delivering large genetic payloads without using viral vectors.