Gene delivery to the lung in vitro – reducing the number of transgenic animals

Dr. Marius Hittinger, Prof. Dr. Claus-Michael Lehr & Dr. Henrik Groß,
PharmBioTec GmbH,
Saarbrücken, Germany


There is an increasing number of newly developed transfection reagents that are promising candidates for gene therapy. With a view to genetically caused lung diseases such as SP-B deficiency or cystic fibrosis, such transfection reagents offer a promising option for a gene therapeutic approach to contribute to the quality of the patient´s life. As there is no cure for diseases like CF currently available, the need for a different approach and the use of gene therapeutics/gene delivery is still high and offers the most promising chances of success. As with every newly developed technique or reagent, a first line test system is needed to show the efficacy of the newly developed reagents.

Animal experiments have become standard in order to investigate the efficacy of gene delivery systems, leading to an ongoing discussion about advantages and limits of alternative in vitro methods. As a consequence, our aim is to establish and to evaluate in vitro models of the lung as tools to study new gene delivery systems to serve as an alternative for animal models and even as a replacement of those studies in the early stage of development. Cell lines and primary human alveolar cells will be investigated with focus on their opportunities to reduce, replace or refine animal experiments according to Russell and Burch.

Fig. 1:
Different in vitro models of the alveolar and bronchial region of the lung will be investigated at the air-liquid interface and at submerged culture conditions. Effects on transfection efficacy as a consequence of the model will be determined.


PharmBioTec GmbH, Saarbrücken, Germany


01/2016 - 04/2017