UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced that the European Medicines Agency (EMA) has granted early ...

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 ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

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, ...

An experimental drug suggests that a 'path is clearly achievable' to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic ...

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Genetic testing plays an important role in cystic fibrosis screening and diagnosis. It can help determine if a person has cystic fibrosis or carries gene mutations that cause it. Other tests can also ...