Able to provide written informed consent. Patients must be able to provide written informed consent Patients must be able to provide written informed consent Able to provide written informed consent Patients must be able to provide written informed consent Patients must be of appropriate mental capacities with sufficient social support so as to be able to complete required study activities (i.e. diet record, etc) and able to provide written informed consent ARM I&II: Patients must be able to provide written informed consent Patients must be able to provide written informed consent Patients must be able to provide written informed consent. Able to provide written informed consent Must be able to provide written informed consent before any screening procedures. Able to provide written informed consent for the trial Able to provide written informed consent. Able to provide written informed consent Subjects must be able to provide written informed consent. Patient able to provide written informed consent; parent or guardian of minor patient able to provide written informed consent Patient able to provide written informed consent for the long-term follow-up gene therapy study: 2006-0676; parent or guardian of minor patient able to provide written informed consent for the long-term follow-up gene therapy study: 2006-0676 Able to provide written informed consent Patients must be able to provide written informed consent Subjects must be able to provide written informed consent Able to provide written informed consent Patient must be able to provide written informed consent Patients must be able to provide written-informed consent Participant must be able to provide written Informed Consent Patients are not able to provide written informed consent to study participation Able to provide written informed consent Able to provide signed, written informed consent Patient able to provide written informed consent. Patient able to provide written informed consent for the long-term follow-up (LTFU) gene therapy study. Patients must be able to provide written informed consent Patients must be able to provide written informed consent Patients must be able to provide written informed consent Subjects or their legal representatives must be able to provide written informed consent. Patients must be able to provide written informed consent Able to provide written informed consent Able to provide written informed consent and to follow protocol requirements. Able to provide informed written consent Subject is able to provide written informed consent. Able to provide informed written consent Patients must be able to provide written informed consent Able to provide written informed consent Subjects must be able to give written consent to the study Able to provide written consent Able to provide written, informed consent Patients must be able to provide written informed consent Patients must be able to provide written informed consent Able to provide written informed consent. Able to provide written informed consent and to follow protocol requirements Subject is able to voluntarily provide written informed consent. Patients must be able to provide written informed consent Able to provide written informed consent Able to provide written informed consent, and agree to practicing 2 forms of birth control during the study Be able to provide written informed consent Able to provide written informed consent Able to provide written informed consent Patients must be able to provide written informed consent Be able to provide written informed consent Able to provide written informed consent Patients must be able to provide written informed consent Patients must be able to provide written informed consent Able to provide written consent Able to provide written consent Patient is able to provide written informed consent prior to study registration Patients must be able to provide written informed consent Patients must be able to provide written informed consent Able to provide written informed consent Able to provide written consent Able to provide written consent Subject and/or guardian is able to provide written informed consent prior to study registration Patient and/or guardian is able to provide written informed consent prior to study registration Patient and/or guardian is able to provide written informed consent prior to study registration Patient and/or guardian is able to provide written informed consent prior to study registration Able to provide written informed consent Patients must be able to provide written informed consent Able to provide written informed consent. Able to provide written consent Able to provide written informed consent Patients must be able to provide written informed consent Patient and/or guardian is able to provide written informed consent prior to study registration Subjects must to able to provide informed written consent prior to study entry Patients must be able to provide written informed consent Must be able to provide a written informed consent Able to provide written informed consent Able to provide written informed consent. Able to provide written informed consent