Gene therapy strategies for idiopathic pulmonary fibrosis: recent advances, current challenges, and future directions

Mitchel J.R. Ruigrok, Henderik W. Frijlink, Barbro N. Melgert, Peter Olinga*, Wouter L.J. Hinrichs

*Bijbehorende auteur voor dit werk

Onderzoeksoutputpeer review

9 Citaten (Scopus)
49 Downloads (Pure)


Idiopathic pulmonary fibrosis (IPF) is a chronic disease in which the lungs become irreversibly scarred, leading to declining lung function. As currently available drugs do not cure IPF, there remains a great medical need for more effective treatments. Perhaps this need could be addressed by gene therapies, which offer powerful and versatile ways to attenuate a wide range of processes involved in fibrosis. Despite the potential benefits of gene therapy, no one has reviewed the current state of knowledge regarding its application for treating IPF. We therefore analyzed publications that reported the use of gene therapies to treat pulmonary fibrosis in animals, as clinical studies have not been published yet. In this review, we first provide an introduction on the pathophysiology of IPF and the most well-established gene therapy approaches. We then present a comprehensive evaluation of published animal studies, after which we provide recommendations for future research to address challenges with respect to the selection and use of animal models as well as the development of delivery vectors and dosage forms. Addressing these considerations will bring gene therapies one step closer to clinical testing and thus closer to patients.

Originele taal-2English
Pagina's (van-tot)483-496
Aantal pagina's14
TijdschriftMolecular Therapy - Methods & Clinical Development
Vroegere onlinedatumjan.-2021
StatusPublished - 12-mrt.-2021

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