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Save messengers -- modified mRNAs open up new therapeutic possibilities
Date:2/7/2011

s shuttle genetic information into cells in a form that can be used directly for the production of therapeutically active proteins. In addition to their potential for the treatment of genetic diseases, they represent an alternative to established protein-based treatment regimes. The researchers demonstrated the efficacy of the latter application in a mouse model. A single intramuscular injection of mRNAs coding for the hormone erythropoetin, which stimulates the differentiation of red blood cells, led to a significant increase in erythrocyte counts four weeks later.

The team also employed a mouse model to show that the technique can effectively be used to treat a genetic disease. For this, they chose a strain with a lethal congenital lung defect, caused by its inability to make surfactant protein B (SP-B). Regular application of mRNA for SP-B, as an aerosol, to the lungs of the mutant mice restored lung function, and the effect persisted for the duration of the study. Rudolph sees great scope for transcript therapy in regenerative medicine and the treatment of metabolic diseases, and hopes to test the efficacy of modified mRNAs in a clinical setting over the next few years.


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Contact: Dr. Carsten Rudolph
Carsten.Rudolph@med.uni-muenchen.de
49-895-160-7711
Ludwig-Maximilians-Universitt Mnchen
Source:Eurekalert

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