Cystic Fibrosis Gene Therapy: Key Questions and Prospects

Cystic fibrosis is a monogenic disorder with significant morbidity and mortality, despite advances in conventional treatment. It is a good candidate for gene therapy and this field has progressed rapidly since the cystic fibrosis transmembrane conductance regulator gene was cloned. We will review the specific questions to address for successful cystic fibrosis gene therapy, such as the extra- and intracellular barriers to airway gene transfer, the target cells and the endpoints to assess efficacy. We will discuss recent advances in viral and nonviral gene transfer agents, delivery techniques and novel strategies to enhance airway gene transfer and expression.