Supplementary Materials

This PDF file includes:

  • Materials and Methods
  • Fig. S1. AAVrh.10hCLN2 vector.
  • Fig. S2. Trajectory planning for gene therapy infusions.
  • Fig. S3. Axial T2 FLAIR (T2 FLAIR) assessment of participants after therapy.
  • Fig. S4. T cell responses to AAVrh.10 capsid and CLN2 transgene.
  • Fig. S5. MRI assessment of the treated CLN2 children versus untreated CLN2 children.
  • Fig. S6. Quantitative MRI assessment of the treated CLN2 children (cohort 1) versus untreated CLN2 children.
  • Fig. S7. Mullen scale quantitation of the rate of decline for cohorts 1 (red, treated) and 2 (blue, control).
  • Fig. S8. Impact of treatment on quality of life as assessed by age-dependent quality of life questionnaires.
  • Fig. S9. Correlation of various parameters to the rate of decline of motor + language of cohort 1.
  • Table S1. CLN2 disease severity clinical rating scales.
  • Table S2. Vector infusion time and operating room surgery and anesthesia duration in cohort 1 participants.
  • Table S3. Vector infusion time and operating room surgery and anesthesia duration in cohort 4 participants.
  • Table S4. CSF nucleated cells.
  • Table S5. Percent volume of the brain with MRI T2 hyperintensity.
  • Table S6. Quality of life questionnaires.
  • Table S7. Coefficient of variation among observers in the CLN2 disease motor + language neurologic rating scale.
  • Table S8. Reproducibility of motor and language assessment.
  • Table S9. Assessments of motor and language parameters for cohort 2.
  • Table S10. Inclusion/exclusion criteria for cohorts 1 and 2.
  • Table S11. Timeline of the clinical study.
  • Table S12. Inclusion/exclusion criteria for cohort 4.
  • References (5868)

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