Crispr Cas 9

Question

Hello,
unfortunately our two daughters (7 years and 7 months) suffer from CF. We are in very good care and both girls are fit and stable.
Some days ago my physician wanted to know something about the health status of our girls. He mentioned also Crispr Cas and was interested in how far the actual state of research is concerning CF.
I had alreday heard about it, however did not pay too much attention to the topic (ethics, no information on usage in the media, no reports, no information from the CF physicians of our daughters)...
My physician, however, has a different opinion on it. Due to a missing intervention into stem cells, therefore the genetic material of a human being, this method would not fall under the ethic law. Only mutated DNA sequences would be "cutted out". Especially in case of herited diseases he regards this genetic scissors to be promising.
However this seems to be very sensibel, if there are not yet any irreparrable damages (that means in case of CF e.g. of the lungs).
I now read about Ulm (city in Germany), however a real Status Quo I could not find.
How is your state of knowledge here concerning the usage of the genetic scissors, chances? We of course do not want to miss anything in case of our girls.
Many thanks for your answer.

Answer

Dear questioner,
the genetic correction done by CRISPR/CAS9 (genetic scissors) is discussed lively in the field of research. The estimate, to perform with this technology in body cells a so-called somatic genetic therapy, is therefore in principle correct.
For CF, this project is however only in the state of research, that means, one can work well with cell-culture models in the culture dish, even the transmission of the technique to the animal model, could not yet be shown for CF (for other illnesses, there are alreday successfull attemps in the animal model).
A great difficulty is to reach the "right cells" - in case of CF these are especially the lung superficial cells of the lower airways. Here one still does not reach this region for sure with the tools (for comparison: cells of the blood, that can be reached comparably better and can also be changed outside the body, can already be changed successfully with somatic genetic therapy).
In summary it is at the moment unfortunately like this: the somatic genetic therapy (also with CRISPR/CAS9) is a research field and still far away from the concrete usage at the CF patient. I have a recommendation for reading:
www.zeit.de/2016/27/emmanuelle-charpentier-crispr-gentechnik

[in German]
This article is well understandable and illuminates the technique very balanced.
With my best regards,
Frauke Stanke

03.04.2017