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An artist’s representation of the human respiratory system with pink airways on a blue and black background.
A Gene Editing System Corrects Cystic Fibrosis Mutation
An optimized version of prime editing technology raises the possibility of a one-time treatment for cystic fibrosis.
A Gene Editing System Corrects Cystic Fibrosis Mutation
A Gene Editing System Corrects Cystic Fibrosis Mutation

An optimized version of prime editing technology raises the possibility of a one-time treatment for cystic fibrosis.

An optimized version of prime editing technology raises the possibility of a one-time treatment for cystic fibrosis.

cystic fibrosis

Dark blue RNA strands with a light blue background.
A CRISPR Tool for Tinkering with the Human Transcriptome
Danielle Gerhard, PhD | Jul 23, 2024 | 5 min read
Human cells repair RNA breaks induced by a programmable CRISPR system, paving the way for temporary treatments for genetic diseases.
An abstract illustration of a DNA helix and human lungs.
A New Delivery System Offers Hope for Cystic Fibrosis
Charlene Lancaster, PhD | May 1, 2024 | 4 min read
CRISPR-carrying lipid nanoparticles enabled researchers to correct a rare nonsense mutation in the lungs of a cystic fibrosis mouse model.
An illustration of a chromosome with a yellow-colored mutation.
Unraveling Rare and Inherited Diseases with Genetic Technologies
Thermo Fisher Scientific | 1 min read
Rare genetic diseases are difficult to diagnose and characterize in the laboratory because of their heterogeneity and global scarcity.
Ferret and DNA composition
Ferreting Out the Causes of Cystic Fibrosis
Danielle Gerhard, PhD | Feb 1, 2024 | 2 min read
Transgenic ferret models ratted out how a rare cell type affects airway function.
The Breakthrough Prize ?Trophy
2024 Breakthrough Prizes in Life Sciences
Danielle Gerhard, PhD | Sep 14, 2023 | 10 min read
This year’s Breakthrough Prizes honor advances in CAR T cancer therapies, cystic fibrosis, and Parkinson’s disease.
Don Ingber engineered cystic fibrosis airway chips that recapitulate patient symptoms.
A New Model of Lung Disease Paves the Way for Personalized Treatments
Nele Haelterman, PhD | May 9, 2022 | 4 min read
Scientists engineered a lung-on-a-chip model from patient cells that mimics cystic fibrosis.
a photo of the packaging for the drug Trikafta
FDA Approves New Cystic Fibrosis Drug
Shawna Williams | Oct 24, 2019 | 1 min read
The treatment, Trikafta, increases lung function in most patients with the disease—but comes with a hefty price tag.
Genetically Modified Viral Cocktail Treats Deadly Bacteria in Teen
Ashley Yeager | May 8, 2019 | 2 min read
Tweaking the genomes of two phages and combining them with a third phage helped to clear a persistent Mycobacterium infection in the patient.
Lack of Diversity in Genetic Datasets is Risky for Treating Disease
Ashley Yeager | Mar 21, 2019 | 6 min read
Certain populations have been historically underrepresented in genome sequencing studies, but the NIH, private clinics, and 23andMe and other companies are trying to fix that.
Image of the Day: Breathe In
Carolyn Wilke | Jan 14, 2019 | 1 min read
Inhalable particles carrying mRNA make mouse lungs glow.
New Lung Cell Identified
Abby Olena, PhD | Aug 1, 2018 | 4 min read
The cell type was discovered via single-cell RNA sequencing of thousands of cells in mouse and human airways and may play a role in cystic fibrosis.
CF Gene Therapy Shows Promise
Jef Akst | Jul 6, 2015 | 1 min read
The results of a Phase 2 trial suggest that delivering normal copies of the gene that causes cystic fibrosis may slow lung decline.
Genome Digest
Jenny Rood | Apr 16, 2015 | 6 min read
What researchers are learning as they sequence, map, and decode species’ genomes
Breathing Life into Lung Microbiome Research
Rina Shaikh-Lesko | Jun 9, 2014 | 4 min read
Although it’s far less populated than the mouth community that helps feed it, researchers increasingly appreciate the role of the lung microbiome in respiratory health.
FDA Approves NGS Tests
Tracy Vence | Nov 21, 2013 | 1 min read
The agency has cleared marketing of four next-generation sequencing devices from Illumina.
The Road Less Traveled
Megan Scudellari | Nov 1, 2012 | 9 min read
First, Aravinda Chakravarti drew a map of how scientists might unravel the genetics of complex disease. Then he blazed the trail.
Cystic Fibrosis Alters Microbiome?
Edyta Zielinska | Sep 28, 2012 | 1 min read
The microbiome of the lung is different in patients with the disease, which causes a thick buildup of mucus that makes breathing difficult.
Novel Cystic Fibrosis Drug Approved
Megan Scudellari | Feb 1, 2012 | 2 min read
After a lightning-fast approval, the first medicine to treat an underlying cause of the disorder hits the market.
Infection Selection
Ruth Williams | Nov 13, 2011 | 2 min read
Scientists track changes in bacterial genomes during a hospital outbreak to discover potential pathogenesis genes.
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