Gene therapy for cystic fibrosis: recent progress and current aims
File(s)
Accepted version
Accepted version
Author(s)
Paul-Smith, MC
Bell, RV
Alton, WE
Alton, EWFW
Griesenbach, U
Type
Journal Article
Abstract
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.
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.
Date Issued
2016-05-06
Date Acceptance
2016-04-18
Citation
Expert Opinion on Orphan Drugs, 2016, 4 (6), pp.649-658
ISSN
2167-8707
Publisher
Taylor and Francis
Start Page
649
End Page
658
Journal / Book Title
Expert Opinion on Orphan Drugs
Volume
4
Issue
6
Copyright Statement
© 2016 Taylor & Francis. This is an Author's Accepted Manuscript of an article published in Expert Opinion on Orphan Drugs, available online at: http://dx.doi.org/10.1080/21678707.2016.1180974
Sponsor
Medical Research Council (MRC)
Cystic Fibrosis Trust
Grant Number
MR/J014699/1
VIA 013
Subjects
Science & Technology
Life Sciences & Biomedicine
Pharmacology & Pharmacy
Cystic fibrosis
gene therapy
lung
viral vector
non-viral vector
TRANSMEMBRANE CONDUCTANCE REGULATOR
LONG-TERM SAFETY
NASAL POTENTIAL DIFFERENCE
CHLORIDE TRANSPORT DEFECT
AIRWAY EPITHELIAL-CELLS
PLURIPOTENT STEM-CELLS
PHASE 2B TRIAL
RECOMBINANT ADENOVIRUS
LENTIVIRAL VECTOR
CFTR GENE
Publication Status
Published