Supplementary Materials

Supplementary Material for:

Sustained Normalization of Neurological Disease After Intracranial Gene Therapy in a Feline Model

Victoria J. McCurdy, Aime K. Johnson, Heather L. Gray-Edwards, Ashley N. Randle, Brandon L. Brunson, Nancy E. Morrison, Nouha Salibi, Jacob A. Johnson, Misako Hwang, Ronald J. Beyers, Stanley G. Leroy, Stacy Maitland, Thomas S. Denney, Nancy R. Cox, Henry J. Baker, Miguel Sena-Esteves, Douglas R. Martin*

*Corresponding author. E-mail:

Published 9 April 2014, Sci. Transl. Med. 6, 231ra48 (2014)
DOI: 10.1126/scitranslmed.3007733

This PDF file includes:

  • Fig. S1. β-Gal distribution in the CNS of GM1 cats after treatment with AAV1.
  • Fig. S2. Clinical progression of AAV1-treated GM1 cats.
  • Fig. S3. β-Gal activity in the CNS and CSF of GM1 cats treated long term.
  • Fig. S4. Hypereosinophilic neurons in the cortex of an AAV-treated GM1 cat.
  • Fig. S5. Hexosaminidase activity in the liver of GM1 cats treated long term.
  • Table S1. Vector copy number in brain, spinal cord, and liver of AAV-treated GM1 cats.
  • Table S2. Vector copy number and β-Gal activity in AAV-treated GM1 cats and their offspring.
  • Legends for videos S1 to S3

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Other Supplementary Material for this manuscript includes the following:

  • Video S1 (.mpg format). Untreated GM1 cat near the humane endpoint (6.6 months of age).
  • Video S2 (.mpg format). Clinically normal GM1 cat after AAV treatment (34.7 months of age).
  • Video S3 (.mpg format). AAV-treated GM1 cat with slight hindlimb gait deficit (39.0 months of age)

[Videos S1 to S3]