Gene editing

Question

Hello,
The last magazine edited by the French CF patient organization indicates that genetic editing techniques, for correcting the genetic material in a relatively simple and inexpensive way, have been developed. They allow to avoid vectors and correct cells directly. Could we have more information? Is it the work done by a team of researchers from Yale? Is it to correct the gene or a mutation in particular? Thank you very much for your answer.

Answer

Hello,

Regarding the technique of "gene editing" or "genomic edition", it comes to "rewrite" the message included on a gene by reintegratation the original genetic formula. This refers to a set of techniques to "cut" the DNA where is an "error" then make a new sequence corresponding to the healthy gene.

Combining this new technology with the work done in the field of stem cells, could be an interesting alternative to gene therapy as envisaged today. It's in this sense that the cells would be "corrected directly" and that "it would avoid vectors".

The research is not there at the moment. It is a line of research which is at the very basic stage, even if the potential therapeutic benefits are very attractive and generate real enthusiasm among researchers in the field.

Regards,
Veronique Marguerite-Heissat, PhD
Deputy Scientific Director
Responsible for basic research and translational
CF French Association "Vaincre La Mucoviscidose"

12.10.2015