AVM are genetic diseases caused by mutations of the genes. In this way, AVM can be addressed with gene therapy. Gene therapy starts with the repairing or replacement of the gene that causes the disease. If this does not work, additional genes will be inserted into the body to address the mutated gene. Danon Disease, the most common form of AVM, could possibly be cured by adeno-associated virus 9 (AAV9)–mediated gene therapy. Clinical studies on mice with the mutated LAMP2 gene have shown that the injection of the normal human LAMP2B gene into Lamp2 knockout mice actually managed to cure the mice from Danon disease. This method could be a permanent solution since the gene would be able to generate the protein needed and normal functions of the protein would be restored. Nevertheless, this method has not yet been trialed on human subjects so it needs further clinical trials to show its validity. The same concept could be applied to other AVM, targeting different gene mutations by introducing the normal version of the mutated gene.
