Gene Therapy for Cystic Fibrosis Paved the Way for the Use of Adeno-Associated Virus in Gene Therapy

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish (US)
Pages (from-to)538-541
Number of pages4
JournalHuman gene therapy
Volume31
Issue number9-10
DOIs
StatePublished - May 2020

Keywords

  • Adeno-associated virus
  • Clinical trials
  • Cystic fibrosis
  • Gene therapy
  • Preclinical testing

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

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