Background: Despite the proof of principle that gene therapy can cure various monogenic
diseases, limited clinical progress has been noted for gene therapy of the respiratory system.
Certain anatomic features of the lungs, along with the suboptimal gene delivery vehicles utilized
up to now, have significantly delayed successful clinical practice. Thus, the need for additional
improvements towards safety and efficacy of the procedure is indispensable.
Objective: The objective of this work was to review the progress and limitations of gene therapy in
the treatment of lung disease with a focus on monogenic disease, chronic obstructive pulmonary
disease and asthma and to present studies that provide a proof of principle that it works in different
model systems and in patients.
Method: A thorough search was performed on the aforementioned topic using Pubmed in order to
identify relevant manuscripts. Several gene therapy studies for monogenic disorders affecting
other organs or systems were also taken into consideration.
Results: A hundred and thirty one papers were included. Inclusion criteria regarded novel gene
transfer technologies of the past decade, as well as publications outlining the pitfalls that precluded
earlier successful implementation of gene therapy for pulmonary diseases.
Conclusion: Current gene transfer protocols and vector design require additional amelioration.
The rapidly evolving and much promising technology of CRISPR/Cas9 might possibly overcome
the hurdles posed to date for effective implementation of gene therapy and become the basis for
the onset of new clinical trials.