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Gene Therapy: Is RNA Better DNA?

When he slashed the acronym CRISPR from “Clustered Regularly Interspaced Short Palindromic Repeats”, the Dutch molecular biologist Ruud Jansen was certainly not sure what kind of career his abbreviation would have – otherwise he would probably have come up with something better. But not only that CRISPR is an imposition for the tongue – hardly anyone understands what is actually behind it. Only one thing is clear: it is a powerful tool in the hands of microbiologists, and there is great hope that it will one day be able to cure genetic diseases.

It sounds so easy: cut out the faulty gene, insert a working one, cured. The first CRISPR therapies for sickle cell anemia and cancer have already made it into clinical studies. They are ex vivo treatments. The cells to be treated are removed from the patient, modified in the laboratory with gene scissors and returned to the patient. So far, these have been cells of the blood-forming system, because getting blood or bone marrow is relatively easy.

Medicine still shies away from using gene scissors directly in the body. After all, the CRISPR / Cas9 system is a really big chunk of protein that comes from bacteria. Our immune system will not simply accept it. In addition, it has to be introduced into the right cells in the body. Even for tiny drug molecules, this is a challenge that is insurmountable in some cases. Finally, CRISPR works well, but not perfectly. The system makes mistakes, overlaps, things can go wrong when repairing the cut DNA, and changes in the genetic make-up cells like to acknowledge the development of cancer. In addition to all of these security concerns, there is the ethical aspect of interfering with the human genome.

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