ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA…
Articles by Patricia Inácio, PhD
A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) —…
Girls with cystic fibrosis (CF) under the age of six have higher levels of a tiny RNA molecule, miR-885-5p,…
Developing a way to destroy persister cells — bacterial cells that enter a dormant phase and are able to resist…
A project aimed at personalized medicine based on the use of organoids, or “mini-organs,” from cystic fibrosis (CF) patients…
Cystic fibrosis carriers — individuals who have certain genetic mutations in one of the copies of the CFTR…
Genetic variations that lower the expression levels of a gene called RNF5 may explain why some people with cystic fibrosis…
Babies diagnosed with cystic fibrosis (CF) through newborn bloodspot screening show better lung function, can better stave off infection…