Supplementary Materials

Supplementary Material for:

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

Martin L. Katz, Luis Tecedor, Yonghong Chen, Baye G. Williamson, Elena Lysenko, Fred A Wininger, Whitney M. Young, Gayle C. Johnson, Rebecca E. H. Whiting, Joan R. Coates, Beverly L. Davidson*

*Corresponding author. E-mail: davidsonbl{at}email.chop.edu

Published 11 November 2015, Sci. Transl. Med. 7, 313ra180 (2015)
DOI: 10.1126/scitranslmed.aac6191

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Representative image of rAAV serotype screening for transduction of canine ependymal cells.
  • Fig. S2. TPP1 activity in the CSF from normal dogs.
  • Fig. S3. Unilateral injection of rAAV.caTPP1 results in bilateral distribution of TPP1 activity and TPP1-immunopositive cortical pyramidal cells.
  • Fig. S4. rAAV.caTPP1 biodistribution.
  • Fig. S5. rAAV.caTPP1 improves astroglial activation and does not induce microglial activation.
  • Fig. S6. Representative transfer membranes used for Western blot of SCMAS and p62 in three brain regions (thalamus, occipital cortex, and cerebellar cortex).
  • Fig. S7. Recombinant TPP1 in peripheral organs of rAAV.caTPP1-treated dogs.
  • Table S1. TPP1-null dachshund dogs used for vector and/or drug infusions.
  • Table S2. Tissue and clinical data.
  • Legends for movies S1 to S3
  • Reference (34)

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Other Supplementary Material for this manuscript includes the following:

  • Movie S1 (.mp4 format). Representative video of the gait of a 9.7-month-old untreated TPP1-null dog (DC024).
  • Movie S2 (.mp4 format). Representative video of the gait of a 9.8-month-old TPP1-null dog 6.3 months after rAAV.caTPP1 injection (DC020).
  • Movie S3 (.mp4 format). Life quality and social interactions of a 17.5-month-old TPP1-null dog (DC020), 14.5 months after rAAV.caTPP1 injection.