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A little hope for baby bubbles. A new gene therapy trial, to treat these children with a disease depriving them of immune defenses and forcing them to live in a sterile bubble from birth, should begin in the fall. (News of 26/07/10)
- In March 1999, a team of French doctors treated for the first time by gene therapy "baby bubbles", children deprived of immune defenses and forced to live in sterile rooms. The results had been very encouraging since today, 7 of these children treated in France who have received this treatment are doing well and lead a normal life.
A new clinical trial in autumn
- Eleven years later, the same team has just received the approval of health authorities for a new clinical trial, which will begin in the fall, for 5 children with the same disease, the DICS-X. This rare genetic disease is characterized by the total absence of defense cells against infections. Today, the only cure is a bone marrow transplant.
Gene therapy, how does it work?
- This gene therapy involves inserting a normal copy of the altered gene into the body of sick children. This gene "drug" is inserted outside the body (ex vivo) into the child's cells through a "viral vector". This vector allows the penetration of the gene into the cell, its insertion into the genome and the production of the defective protein in children. Once this step is completed, the "corrected" cells are reinjected to the patient.