Boucher, University of North Carolina Cystic Fibrosis-Pulmonary Research and Treatment Center. The cystic fibrosis transmembrane regulator (CFTR) gene was isolated as the cause of cystic fibrosis (CF) ...
The company’s pipeline includes VX-522, a CFTR mRNA therapeutic designed to treat the underlying cause of CF, which is in Phase 1 clinical trial; VX-548, a non-opioid medicine for the treatment ...
The epigenetic state of chromatin, gene activity, and chromosomal positions are interrelated. A research team from the IPK ...
A research team has identified the core gene expression networks regulated by key proteins that fundamentally drive phenomena such as cancer development, metastasis, tissue differentiation from stem ...
In living organisms, protein molecules with different chemical structures called proteoforms, are produced from a single gene ...
The FDA approved a wide range of therapies in 2024, including many new molecular entities and biological products.
A research team at KAIST has identified the core gene expression networks regulated by key proteins that fundamentally drive ...
Learn the facts on our page, Dispelling Misconceptions About Cystic Fibrosis. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein ...
Gene expression is the generation of a functional gene product from the information encoded by a gene, through the processes of transcription and translation. Gene products are often proteins ...
An in vivo base editing approach targeting the PRNP gene led to a 52% increase in the lifespan of mouse models inoculated with the most common sporadic and genetic types of human pathogenic prion ...