Dear Friends and Colleagues:
My last short research report about the state of exon 51 skipping, which I sent last year to you and about 1,200 other Duchenne families and scientists, was supposed to be my last one. But when the journal Science declared at the end of last year that CRISPR/Cas9 was the “Breakthrough in Genome Editing” in 2015, I realized that it would not take long until this new technique would be applied to finding a new therapy for Duchenne muscular dystrophy. Then, at the end of January, when my Rotary Club in the Black Forest here in Germany needed urgently someone to give a presentation at one of our meetings, I thought, that this new gene technique would be a good subject to talk about. After only a few days for preparation, about 30 power-point slides were ready and I could tell my Rotarian friends why CRISPR/Cas9 is so important in the fight against Duchenne and other hereditary diseases.
Afterwards, I translated my slides into English and after some modifications, here they are enclosed for you and all other English speaking Duchenne families, whose addresses are on my e-mailing lists, because you will really be interested in this new and highly promising technique. Please open the enclosed file with your power-point program and look at the slides with the screen presentation program then you can see all the animations. For those who have not the power-point program on their computer, I have enclosed the slides also as a pdf file. But you will then not be able to see the animations.
If you wish, you can also show my slides to your friends, perhaps even after a translation into another language. You have my permission, just mention my name and if you have a translation, send it to me, too. You should also know that I have shown my slides to Professor Annemieke Aartsma-Rus at the University in Leiden, the Netherlands, to make sure that I did not make any mistakes. Her most important comment was to say that although the CRISPR technique could already help mdx mice impressively, it will take a while until it will work also so positively for Duchenne boys who are much larger than a mouse. After all, the CRISPR system contains a rather large protein which has to be transported into the muscle tissue by harmless adeno viruses. And such protein-transfer techniques cannot yet be applied routinely in Duchenne boys. Therefore, work on exon skipping and the many other Duchenne therapeutic approaches have to continue.
I hope you will like to see that research continues in spite of so many difficulties. Whether I will be able to write another report with better news in a year or two is not certain. After all, I will be 86 in April, and it becomes more and more difficult getting along with my growing health problems, mainly caused by a polyneuropathy.
I am sending you all my best regards and remain your old friend Guenter.
Dr. Guenter Scheuerbrandt
26 February 2016
I just saw in yesterday’s of “Nature” the article “Should you edit your children’s genes?” (enclosed) . Please read this and tell me whether you believe like I do that CRISPR should be used to change the damaged dystrophin gene of a Duchenne boy back to normal and just get rid of his disease as soon as this technique is able to do this. GS