Supplementary Materials

Supplementary Material for:

Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses

Thomas Kirkegaard,* James Gray, David A. Priestman, Kerri-Lee Wallom, Jennifer Atkins, Ole Dines Olsen, Alexander Klein, Svetlana Drndarski, Nikolaj H. T. Petersen, Linda Ingemann, David A. Smith, Lauren Morris, Claus Bornæs, Signe Humle Jørgensen, Ian Williams, Anders Hinsby, Christoph Arenz, David Begley, Marja Jäättelä, Frances M. Platt

*Corresponding author. Email: tkj{at}orphazyme.com

Published 7 September 2016, Sci. Transl. Med. 8, 355ra118 (2016)
DOI: 10.1126/scitranslmed.aad9823

This PDF file includes:

  • Fig. S1. rHSP70 uptake in NPC patient fibroblasts and lymphocytes.
  • Fig. S2. rHSP70 in Npc1−/− mouse model pilot study and manual behavior quantification.
  • Fig. S3. GSL and behavioral analyses of rHSP70-treated Npc1−/− mice.
  • Fig. S4. Antidrug antibody responses against rHSP70.
  • Fig. S5. In vitro analysis of activated HSF1 (pSer326 HSF1).
  • Fig. S6. Effect of arimoclomol on lysosomal accumulation in NPDA and MLD.
  • Fig. S7. Arimoclomol increases survival of Npc1−/− mice.
  • Fig. S8. Plasma and thyroid gland distribution of 125I-rHSP70.
  • Legends for movies S1 to S4

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Other Supplementary Material for this manuscript includes the following:

  • Movie S1 (.avi format). Uptake and effect of rHSP70 on the endolysosomal system in NPD primary patient fibroblasts.
  • Movie S2 (.avi format). Endolysosomal dynamics in NPD primary patient fibroblasts.
  • Movie S3 (.mp4 format). Video of gait analysis of PBS-treated control Npc1−/− mice.
  • Movie S4 (.mp4 format). Video of gait analysis of rHSP70-treated Npc1−/− mice.