Researchers at the University of Trento have developed a CRISPR-based adenine base editing therapy that repaired the ...
The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current CF therapies.
A cell-penetrating nanobody repairs misfolded CFTR proteins in cystic fibrosis cell models, restoring function and boosting ...
A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...
Chronic lung inflammation in cystic fibrosis (CF) often persists even after treatment with newly-approved gene therapies or small molecule CFTR modulators—an unresolved clinical paradox. A new study ...
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Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...
Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...
The CFTR gene was identified in 1989, but few companies jumped at the chance to use the information to develop new therapies, because the market was perceived as too small. It is therefore somewhat ...
Inside the deadly disease confounding medicine, researchers piecing together clues and the breakthrough that meant survival ...
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