From disease mechanisms to novel therapies and back

Never before has CF care and research been so filled with hope. Understanding of the pathogenesis cascade of this lethal disorder is still not complete, but since the early days it has become clear that an effective therapy needs to tackle the basic defect – either by correcting the gene or by repairing either CFTR mRNA or protein [1].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research