Genforschung und Gentherapie im Zeitalter der Genom- und Epigenom-Editierung

Genome editing based on the CRISPR/Cas system has revolutionized genetic research and seems to promise safe gene therapy applications on soma and germline cells. However, the CRISPR/Cas system can also be used for epigenome editing which does not change the DNA sequence as such, but »merely« modifies the expression of the gene sequences. Moreover, changes on the epigenetic level cannot be passed on to the next generation and, as a result of this reversibility, prove to be less invasive. Epigenome editing will probably be used primarily to treat diseases that are caused by defective gene expressions (imprinting disorders). In addition, other fields of application - such as certain monogenetic or viral diseases - are already emerging; in future, they could eventually be treated alternatively with both genome and epigenome editing. Germline cells, on the other hand, overwrite their epigenetic state; epigenome editing therefore seems less suited for germline interventions. However, as research on the epigenome is just beginning to gain momentum, it cannot be ruled out for the future that epigenetic interventions will also be possible in this respect.