{"id":6226,"date":"2020-06-02T09:47:17","date_gmt":"2020-06-02T07:47:17","guid":{"rendered":"https:\/\/www.institut-biotherapies.fr\/?p=6226"},"modified":"2020-06-09T09:57:31","modified_gmt":"2020-06-09T07:57:31","slug":"a-genethon-team-has-succeeded-in-inhibiting-the-immune-response-linked-to-aav","status":"publish","type":"post","link":"https:\/\/www.institut-biotherapies.fr\/en\/2020\/06\/02\/a-genethon-team-has-succeeded-in-inhibiting-the-immune-response-linked-to-aav\/","title":{"rendered":"A Genethon team has succeeded in inhibiting the immune response linked to AAV"},"content":{"rendered":"

\"UneA research team from Genethon, in collaboration with teams from CNRS\/Inserm and from the biotechnology company Spark Therapeutics, announced in Nature Medicine<\/a> that it has succeeded in inhibiting the immune response induced by AAV antibodies present as a result of natural immunity or following gene therapy, thanks to the IdeS enzyme. This result opens up new therapeutic prospects and the possibility of treating more patients.<\/strong><\/p>\n

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Gene therapy consists of injecting a therapeutic gene into an organism using a vector, a “means of transport” able to cross all biological barriers through the cell and into the nucleus. The most commonly used vectors are derived from viruses, such as AAVs (adeno-associated viruses), used particularly in gene therapy targeting the muscles, liver, eyes, etc. In many cases, once it comes into contact with this virus, the body develops immunoglobulins (IgG) known to be neutralising, i.e. specific antibodies that inhibit AAVs. It is considered that between 30% and 50% of humans are naturally immunized against most AAVs used for therapeutic purposes. As a result, a large number of patients cannot currently benefit from AAV gene therapy. Moreover, a first injection of an AAV leads to an immune response against the vector, precluding any subsequent AAV gene therapy.<\/p>\n

Inhibiting the immune response thanks to the IdeS enzyme<\/h2>\n

Overcoming this impediment is an essential factor in treating the maximum number of patients. Therefore, the teams lead by Giuseppe Ronzitti, a Genethon researcher, S\u00e9bastien Lacroix-Desmazes, a CNRS-Inserm researcher, and Federico Mingozzi from the biotechnology company Spark Therapeutics, have used animal models to test the efficacy of the IdeS enzyme<\/strong>, an endopeptidase that naturally reduces the action of antibodies, in order to neutralise the immune response due to IgG immunoglobulins.
\nThe study was carried out in two steps:<\/p>\n