title: A possible advance in arterial gene therapy for aortic complications in the Marfan syndrome by local transfer of an antisense Mg-dependent hammerhead ribozyme.
abstract: 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.
authors: Magnesium Research, Neuilly-sur-Seine, France.
authors: J
authors: J
authors: Durlach
mesh_terms: D000483:Alleles; D017130:Angioplasty; D000818:Animals; D001011:Aorta / Q000002:abnormalities / Q000378:metabolism*; D002478:Cells, Cultured; D000071838:Fibrillin-1; D000071837:Fibrillins; D005347:Fibroblasts / Q000378:metabolism; D015316:Genetic Therapy / Q000379:methods*; D006331:Heart Diseases / Q000150:complications*; D006801:Humans; D020136:Hydrogel, Polyethylene Glycol Dimethacrylate; D008274:Magnesium / Q000378:metabolism*; D008382:Marfan Syndrome / Q000628:therapy*; D008840:Microfilament Proteins / Q000235:genetics; D016376:Oligonucleotides, Antisense / Q000235:genetics*; D016337:RNA, Catalytic / Q000235:genetics*; D011817:Rabbits; D054392:Adipokines
chemical_list: D000071838:Fibrillin-1; D000071837:Fibrillins; D020136:Hydrogel, Polyethylene Glycol Dimethacrylate; D008274:Magnesium; D008840:Microfilament Proteins; D016376:Oligonucleotides, Antisense; D016337:RNA, Catalytic; C000605722:FBN1 protein, human; D054392:Adipokines; C427204:hammerhead ribozyme