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 ...

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 ...

Most countries have regulations in place preventing babies being born after gene-editing, but the incident ... copies of a single gene, such as cystic fibrosis Rigorous checks at every stage ...