{"id":502372,"date":"2026-02-25T04:19:08","date_gmt":"2026-02-25T04:19:08","guid":{"rendered":"https:\/\/www.newsbeep.com\/au\/502372\/"},"modified":"2026-02-25T04:19:08","modified_gmt":"2026-02-25T04:19:08","slug":"advancing-gene-therapy-for-cystic-fibrosis","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/au\/502372\/","title":{"rendered":"Advancing Gene Therapy for Cystic Fibrosis"},"content":{"rendered":"

Original story from University of California, Los Angeles (UCLA; CA, USA).<\/a><\/p>\n

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 and establishing a potential new path toward mutation-agnostic gene therapy for inherited lung diseases.<\/p>\n

The study shows that lipid nanoparticles \u2013 tiny fat-based particles widely used to deliver mRNA vaccines \u2013 can be engineered to carry the complex molecular cargo required for precise insertion of a large full-length gene into the genome without using viral vectors.<\/p>\n

\u201cThis work shows that we can package everything needed for precise gene insertion into a single, non-viral delivery system,\u201d said Steven Jonas, senior author of the study and a member of the UCLA Broad Stem Cell Research Center. \u201cThat\u2019s a critical step toward developing gene therapies that can work across many different disease-causing mutations.\u201d<\/p>\n

Cystic fibrosis is caused by mutations in a single gene, the cystic fibrosis transmembrane conductance regulator, or CFTR, which encodes a channel that helps move chloride and water across the surface of airway cells. When the channel does not function properly, mucus in the lungs becomes thick and sticky, trapping bacteria and leading to chronic infections and progressive lung damage.<\/p>\n

Although highly effective drugs known as CFTR modulators have transformed care for many people with cystic fibrosis, about 10% of patients produce little or no CFTR protein at all, leaving nothing for those drugs to act on.<\/p>\n

\u201cFor those patients, gene therapy isn\u2019t just an improvement \u2013 it\u2019s really the only option,\u201d said Brigitte Gomperts, co-author of the study and associate director of translational research at the stem cell center. \u201cYou have to give the cell the ability to make the protein in the first place.\u201d<\/p>\n

\"\"The model system transforming pulmonary research: an interview with Darrell Kotton<\/a><\/p>\n

In this interview, explore the unique complexities of developing targeted therapies for diseases of the lungs\u00a0\u2013 one of the most resilient and protected organs.<\/p>\n

A new way to deliver a complete gene<\/p>\n

Since there are over 1,700 different mutations in the CFTR gene that can cause cystic fibrosis, the team looked to develop a universal approach that could correct any of these errors in a single edit rather than individually.<\/p>\n

Most experimental gene therapies rely on viral vectors to deliver genetic material into cells. While powerful, viral approaches can be costly to manufacture, limited in the amount of genetic material they can carry and difficult to administer more than once because the immune system can recognize and react to them.<\/p>\n

In this study, the UCLA team instead used lipid nanoparticles as a non-viral delivery system. The particles were engineered 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.<\/p>\n

\u201cGetting all of that into a single particle \u2013 especially a gene as large as CFTR \u2013 is something that hadn\u2019t been shown before,\u201d said Ruth Foley, the study\u2019s first author and a recent PhD graduate from the Jonas lab at UCLA. \u201cIf you can solve the \u2018big gene\u2019 problem, it opens the door for a lot of other diseases as well.\u201d<\/p>\n

The researchers tested the system in lab-grown human airway cells carrying a severe cystic fibrosis mutation that does not respond to existing drugs. The nanoparticles successfully delivered a healthy CFTR gene into about 3\u20134% of the cells.<\/p>\n

Despite that relatively small fraction of corrected cells, the treatment restored between 88% and 100% of normal CFTR channel function across the cell population.<\/p>\n

The researchers say the strength of that recovery reflects not just where the gene was inserted, but how it was engineered.<\/p>\n

The replacement CFTR gene was designed to maximize protein production once it entered the cell, enabling even a small number of corrected cells to have an outsized effect.<\/p>\n

That gene design \u2013 known as codon optimization \u2013 was developed by collaborators in [Doctor] Donald Kohn\u2019s lab at UCLA and boosts CFTR protein production without altering the protein itself.<\/p>\n

Toward durable, one-time therapies<\/p>\n

Unlike approaches that deliver messenger RNA \u2013 which must be repeatedly re-dosed \u2013 the new strategy inserts the corrected gene directly into the genome, potentially allowing cells and their descendants to continue producing functional CFTR over time.<\/p>\n

For long-term benefit, however, gene editing ultimately needs to reach airway stem cells, which sit deep within the lung\u2019s protective lining and regenerate the airway throughout a person\u2019s life.<\/p>\n

\u201cThese stem cells are long-lived and constantly regenerate the airway,\u201d said Gomperts, who is also a professor of pediatrics and pulmonary medicine at the David Geffen School of Medicine at UCLA. \u201cIf you can correct them, you could, in theory, have a lasting source of healthy cells.\u201d<\/p>\n

Reaching those cells remains one of the biggest challenges ahead. The airway is designed to block foreign particles, and in patients with cystic fibrosis, thick mucus creates an additional barrier.<\/p>\n

\u201cThis paper is a proof of concept,\u201d said Jonas, who is also an assistant professor of pediatrics at the medical school and a member of the California NanoSystems Institute<\/a> (CA, USA). \u201cIt shows that we can package and deliver the right genetic cargo. The next challenge is getting it to the right cells in the body.\u201d<\/p>\n

A platform with broader implications<\/p>\n

Because lipid nanoparticles are modular and do not rely on viral components, the approach could be more flexible, scalable and potentially more affordable than traditional gene therapies.<\/p>\n

\u201cThis kind of platform gives you room to iterate,\u201d Foley said. \u201cIf you need to re-dose or adapt the cargo for a different disease, you\u2019re not starting from scratch.\u201d<\/p>\n

Beyond cystic fibrosis, the researchers say the strategy could be applied to other genetic lung diseases \u2013 and potentially conditions in other tissues \u2013 caused by large genes with many possible mutations.<\/p>\n

\u201cFor patients who currently have no effective treatments,\u201d Gomperts said, \u201cthis kind of work represents hope \u2013 not because it will be ready tomorrow, but because it shows a path forward.\u201d<\/p>\n

This article has been republished from the following\u00a0materials<\/a>. Material may have been edited for length and house style. For further information, please contact the cited source. Our press release publishing policy can be accessed\u00a0here<\/a>.<\/p>\n","protected":false},"excerpt":{"rendered":"Original story from University of California, Los Angeles (UCLA; CA, USA). UCLA researchers have developed a lipid nanoparticle-based…\n","protected":false},"author":2,"featured_media":502373,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[10],"tags":[64,63,1619,5279,137],"class_list":["post-502372","post","type-post","status-publish","format-standard","has-post-thumbnail","category-health","tag-au","tag-australia","tag-crispr","tag-cystic-fibrosis","tag-health"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts\/502372","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/comments?post=502372"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/posts\/502372\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/media\/502373"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/media?parent=502372"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/categories?post=502372"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/au\/wp-json\/wp\/v2\/tags?post=502372"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}