Modulator pills transformed cystic fibrosis care. But the fight for a cure is not over — so long as research remains funded.

After false starts and incremental gains, scientists crack the problem that made breathing impossible. Will answers come fast ...

A systematic review of 52 scientific papers submitted to a world-leading clinical genetics journal from multiple scientists ...

Antiverse, an AI-driven techbio company designing therapeutic antibodies for the most challenging disease targets, today announced the close of a $9.3 million Series A financing, led by Soulmates ...

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 ...

RESEARCHERS at University California, Los Angeles (UCLA) have developed a lipid nanoparticle (LNP)-based system capable of delivering the full cystic fibrosis transmembrane conductance regulator (CFTR ...

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 ...

Objectives To determine the diagnostic yield of cystic fibrosis (CF) using a two-tiered genetic testing approach. Although newborn screening includes CF, this typically only covers a selection of ...

Cystic fibrosis is a rare genetic disease that affects nearly 40,000 adults and children in the United States. In people with CF, mutations in the CFTR gene result in a buildup of thick mucus in ...

Creative Commons (CC): This is a Creative Commons license. Attribution (BY): Credit must be given to the creator. The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, crucial for ...

Advanced biotechnology repurposes two bacterial immune systems to correct large stretches of DNA. Human cells that have been edited with the new retron-based gene editing technology. Orange dots mark ...