[1] Hosp Clin Porto Alegre, Serv Genet Med, BR-90035003 Porto Alegre, RS, Brazil
[2] Univ Fed Rio Grande do Sul, Postgrad Program Genet & Mol Biol, Dept Genet, BR-90046900 Porto Alegre, RS, Brazil
[3] Johannes Gutenberg Univ Mainz, Childrens Hosp, D-6500 Mainz, Germany
[4] Shire Human Genet Therapies Clin Res & Dev, Cambridge, MA USA
[5] Hosp Sick Children, Toronto, ON M5G 1X8, Canada