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Abstract
Gene therapy holds promise for the treatment of a range of inherited pulmonary disorders. However, efficient delivery and expression of the therapeutic transgene at levels sufficient to result in phenotypic correction of the diseased state has proved elusive. This review focuses on the development of gene delivery strategies for the lungs. One of the principal prerequisites for successful gene therapy is the delivery of gene vectors to the target area within a tissue and to target cells within that area. Physical and biological targeting of the gene vectors and its application in various models is discussed. Subsequently, both viral and non-viral vectors are addressed with respect to their transfection efficiency in different lung cells, the longevity of expression and their immunogenicity. Also, the various methods for pulmonary gene delivery are evaluated for their merits and limitations.
Acknowledgement
This work was supported by the German Federal Ministry of Education and Research, grants 01GU0616, 13N8539, BioFuture (0311898), LMUexcellent (Investitionsfonds) and “Magnetoselectofection” (FP6 LSH-PL-019038).