Journal
HUMAN GENE THERAPY
Volume 31, Issue 9-10, Pages 538-541Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/hum.2020.046
Keywords
gene therapy; cystic fibrosis; adeno-associated virus; preclinical testing; clinical trials
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Shortly after the cystic fibrosis (CF) gene was identified in 1989, the race began to develop a gene therapy for this condition. Major efforts utilized full-length cystic fibrosis transmembrane conductance regulator packaged into adenovirus, adeno-associated virus (AAV), or liposomes and delivered to the airways. The drive to find a treatment for CF based on gene therapy drove the early stages of gene therapy in general, particularly those involving AAV gene therapy. Since general overviews of CF gene therapy have already been published, this review considers specifically the efforts using AAV and is focused on honoring the contributions of Dr. Barrie Carter.
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