In their 2019 paper, Liu’s team used prime editing to alter ... to the development of genetic therapy treatments for those with rarer CF mutations too,” said Lucy Allen, a lung biologist and research ...

The Core maintains a repository of over 50 constructs containing CFTR and other relevant mutations, together with gene editing and other molecular tools useful to the CF community. These include ...

honed on the rare single-gene disorders, such as Huntington disease (HD) and cystic fibrosis (CF), which dominated the field in the last century (see Genetic Testing Timeline). "Geneticists today are ...

Single gene disorders, like Huntington’s disease and cystic fibrosis, actually follow Mendelian inheritance patterns. The PKU-associated enzyme deficiency was determined biochemically in the ...