Gene therapy for cystic fibrosis: recent progress and current aims

Introduction: Since identification of the disease causing gene over 25 years ago, cystic fibrosis (CF) has been at the forefront of gene therapy research. Despite initial optimism, CF gene therapy has proven considerably more challenging than initially anticipated. However, research conducted over the past two decades has clarified the strength and weaknesses of viral and non-viral gene transfer agents for CF gene therapy. Areas covered: The older literature related to CF gene therapy has been reviewed in many publications and we will, therefore, restrict this review to a brief description and discussion of the key lessons learnt, instead focusing on more recent progress in the field which was identified through literature searches. This review will summarize research leading up to the recent pivotal proof-of-concept study showing that non-viral gene therapy can stabilize the decline of lung function in CF patients and also highlight recent advances in viral vector development which may overcome problems related to loss of efficacy on repeated administration. Expert opinion: The demonstration that gene therapy can stabilize CF lung disease is an important milestone in gene therapy.