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New Gene Editing Approach Offers Hope for Cystic Fibrosis Patients
The system restored 88%–100% of CFTR channel function in 3%–4% of airway cells, offering a mutation-agnostic therapy for cystic fibrosis patients with diverse gene mutations.
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3 Articles
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Newswise
NewswiseNanoparticle-based gene editing could expand treatment options for cystic fibrosis
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.
New gene editing approach offers hope for cystic fibrosis patients
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.
·United States
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