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Gene therapy: GL67A

Question
Hello,

The results published in The Lancet Respiratory Medicine by British researchers are positive although modest ... and this news delights us! Could you tell me if, contrary to the protein therapy, gene therapy would be beneficial for all patients or the type of mutation also will affect the outcome? Also, do you know which mutations were carried by the patients who tested the GL67A ?
Thank you in advance for your reply.
Answer
Hello,

Yes, the gene therapy approach has the advantage of being able to benefit patients regardless of the mutations since the corrected transferred gene is added to the genes present in the native genetic code. The publication of the Lancet also indicates that the type of mutation was not part of the inclusion criteria. Mutations of patients in this study are not indicated in the publication in the Lancet that you can view by clicking the following link:

https://www.google.fr/url?sa=t&rct=j&q=&esrc=s&source=web&cd=1&sqi=2&ved=0CCEQFjAAahUKEwjd_fzVvenGAhUjCdsKHc8kDCE&url=http%3A%2F%2Fwww.thelancet.com%2Fpb%2Fassets%2Fraw%2FLancet%2Fpdfs%2FS2213260015002453.pdf&ei=4sqsVd2EAaOS7AbPybCIAg&usg=AFQjCNFSgymLqkd3zzI6eqtTC37U9dsk2A&sig2=1RdV_2LVkIzpk8BRTtODPw&bvm=bv.98197061,d.ZGU

Hope this answer can help.
Best wishes
Gilles RAULT, MD, Roscoff CF Center
01.08.2015