CHARITÉ – UNIVERSITÄTSMEDIZIN BERLIN Cystic fibrosis remains an incurable genetic disorder which impairs lung function and significantly reduces life expectancy. A new combination drug therapy which addresses the disorder’s underlying defects offers a promising new treatment approach. The use of this therapy had previously been limited to adolescents and adults. Designed to meet the highest...
Tag: <span>cystic fibrosis</span>
A new treatment approach for cystic fibrosis
by Cold Spring Harbor Laboratory Identification of NMD-inhibiting ASOs and assessment of their specificity. a Schematic of NMD reporters. The numbers show the CFTR exons in the NMD reporters. The red asterisk (*) indicates the location of the W1282X mutation. b Schematic of ASO screening. 19 MOE-PS-modified 15-mer ASOs (yellow and magenta bars) were designed to cover the presumptive EJC binding sites...
Drug discovery approach reveals why drugs for cystic fibrosis fall short
by The Scripps Research Institute Scripps researchers analyzed many genetic variants associated with cystic fibrosis across the population to determine how the CFTR protein’s functional structure changes with disease and therapeutics. Credit: Scripps Research To cure rare genetic diseases, from cystic fibrosis to Niemann-Pick, scientists at Scripps Research have turned to a computational approach usually...
Stem cell cloning experts unraveling cystic fibrosis
UNIVERSITY OF HOUSTON IMAGE: WA XIAN AND FRANK MCKEON IN THE DEPARTMENT OF BIOLOGY AND BIOCHEMISTRY AND THE STEM CELL CENTER AT UNIVERSITY OF HOUSTON, ARE EXAMINING PRO-INFLAMMATORY STEM CELL VARIANTS IN CYSTIC FIBROSIS AND DEVELOPING THERAPEUTIC COMBINATIONS THAT SELECTIVELY TARGET THEM IN THE CF LUNG, WHILE SPARING THE NORMAL CELLS NEEDED FOR REGENERATIVE REPAIR....
Cystic fibrosis: Restoring airway integrity
UNIVERSITÉ DE GENÈVE IMAGE: HYDRATION RESTORES THE AIRWAY SURFACE SEAL. IN TURQUOISE, THE JUNCTIONS BETWEEN THE EPITHELIAL CELLS (BLUE). CREDIT: UNIGE – LABORATORY OF PROF. MARC CHANSON Cystic fibrosis is a rare genetic disease which can cause very serious symptoms. In particular, patients suffer from chronic bacterial infections that can lead to respiratory failure. It...
Study finds complex relationships between bacteria and markers of lower airway infection and inflammation in cystic fibrosis
ANN & ROBERT H. LURIE CHILDREN’S HOSPITAL OF CHICAGO The lower airways of patients with cystic fibrosis (CF) have unique biochemical features that correlate with the complex communities of lung bacteria typical of this disease, according to a multicenter study led by researchers from Ann & Robert H. Lurie Children’s Hospital of Chicago. These findings...
Novel therapy could help people with asthma, COPD, cystic fibrosis and cancer-related lung disease
UNIVERSITY OF TEXAS M. D. ANDERSON CANCER CENTER HOUSTON ― A multicenter research team co-led by The University of Texas MD Anderson Cancer Center developed the first drug to treat the uncontrolled secretion of mucins in the airways, which causes potentially life-threatening symptoms in millions of Americans with asthma, chronic obstructive pulmonary disease (COPD) and cystic fibrosis...
Researchers find trove of proteins that may influence cystic fibrosis
by University of Toronto “Clubbing” of the fingers is a classic features of Cystic Fibrosis, although not present in many patients. Credit: Jerry Nick, M.D./ Wikipedia Researchers at the University of Toronto have identified hundreds of new proteins that could play a role in cystic fibrosis, and which may shed light on why some patients...
Editing RNA to fix protein problems in cystic fibrosis
by Cold Spring Harbor Laboratory Credit: Pixabay/CC0 Public Domain The process of generating proteins from genes is akin to a factory, where workers follow a set of instructions that, ideally, are effective and clear. But for some people who suffer from cystic fibrosis (CF), one of their genes has a mutation that results in confusing...
Insights into a cystic fibrosis treatment may herald a novel class of drugs
Protein misfolding is a likely culprit in many degenerative disorders. Cystic fibrosis, for instance, is caused by mutations in the CFTR gene that prevent the eponymous protein from assuming its proper configuration. Mutations that impair how other proteins fold have been linked to Alzheimer’s, Parkinson’s, and Huntington’s disease. Now, a new study demonstrates that drugs...