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A possible advance in arterial gene therapy for aortic complications in the Marfan syndrome by local transfer of an antisense Mg-dependent hammerhead ribozyme.

Aortic dissection and rupture make the Marfan Syndrome (MFS), one of the most highly lethal genetic condition, but these events are preceded by simple aortic root dilatation over a long period. A hydrogel coated angioplasty balloon might constitute a possible aortic vector to locally deliver an antisense Mg-dependent hammerhead ribozyme capable to control specifically the genetic alteration of fibrillin 1 responsible for the connective tissue disorder of MFS.