Concepedia

Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease

Victoria J. McCurdy, Hannah E. Rockwell, Julian Arthur, Allison M. Bradbury, Aime K. Johnson, Ashley N. Randle, Brandon L. Brunson, Misako Hwang, Heather Gray‐Edwards, Nancy E. Morrison,

Gene Therapy · 2014 · 79 citations · 43 references

DOI

Concepts

References

43

A lysosome‐to‐nucleus signalling mechanism senses and regulates the lysosome via mTOR and TFEB

Carmine Settembre, Roberto Zoncu, Diego L. Medina et al. · The EMBO Journal · 2012 · 1.9K citations · Full text

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A hypothesis for I-cell disease: Defective hydrolases that do not enter lysosomes

Scot Hickman, Elizabeth F. Neufeld · Biochemical and Biophysical Research Communications · 1972 · 446 citations

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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Steven J. Gray, Sahana Nagabhushan Kalburgi, Thomas J. McCown et al. · Gene Therapy · 2013 · 372 citations

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Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

Lluı́s Samaranch, Ernesto A. Salegio, Waldy San Sebastián et al. · Human Gene Therapy · 2011 · 297 citations

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Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease

Jean Pyo Lee, Mylvaganam Jeyakumar, Rodolfo Gonzalez et al. · Nature Medicine · 2007 · 289 citations · Full text

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