We applied a strategy of serial screening steps to 45 patients with congenital absence of the vas deferens and characterized cystic fibrosis transmembrane conductance regulator gene mutations in ...

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for the treatment of cystic fibrosis in patients with at least one F508del mutation or another treatment-responsive mutation in the CFTR gene. The fixed-dose triple-combination therapy ...

Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with ...

In the 1980s, the discovery of the CFTR gene and mutations that cause CF further solidified the role of the sweat test in ...

Sionna Therapeutics has closed its initial public offering, raising $191 million for its pipeline of therapies for cystic ...

For this, they used a mouse line that carries a mutation in the CFTR gene. Intravenous injections of SORT LNPs carrying a gene editor targeted to this mutation resulted in its correction in almost ...

The CFTR Protein Distribution Core offers high-quality, full-length recombinant cystic fibrosis transmembrane conductance regulator (CFTR) proteins expressed in and purified from mammalian cells. Wild ...

in addition to maintenance of wild-type Sprague Dawley strain for additional use Generation and procurement of novel relevant CF animal models to understand newly developed CFTR modulators, and to ...

Take cystic fibrosis. It’s caused by a defect in the CFTR gene. But you need two copies of the defective gene to actually get the disease. So, if you only have one defective copy, you’re ...

View Full Profile. Learn about our Editorial Policies. In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease and Tay-Sachs disease. Liu explained ...