Sionna Therapeutics today announced the official launch of the company and the closing of a $111 million Series B financing. The round was led by OrbiMed with participation from funds advised by T.

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 ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

Chemical modulation of histone deacetylase (HDAC) activity by HDAC inhibitors (HDACi) is an increasingly important approach for modifying the etiology of human disease. Loss-of-function diseases arise ...

Please provide your email address to receive an email when new articles are posted on . Challenged urine bicarbonate excretion provided a simple and safe quantification of cystic fibrosis ...

(Memphis, Tenn.—March 22, 2023) Scientists at St. Jude Children’s Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis ...

The Gene Expression Core was founded in 1994 and promotes translational, multidisciplinary CF research by furnishing expertise concerning recombinant CFTR and other proteins relevant to CF disease ...

Cystic fibrosis (CF) is the most common inherited metabolic disease in Europe. More than 8,000 people in Germany are affected ...

Scientists at St. Jude Children's Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis transmembrane conductance regulator ...

The cystic fibrosis transmembrane conductance regulator has been studied for years but the new efforts have yielded important insights. Scientists at St. Jude Children's Research Hospital and ...