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Gateway to the diseased brain

Nature Medicine volume 15, pages 1123–1124 (2009)Cite this article

The blood-brain barrier constitutes a major obstacle to effective treatment of diseases affecting the central nervous system. A new strategy to target specifically the endothelium of diseased brain may allow the development of more effective gene-based therapies (pages 1215–1218).

Lysosomal storage disorders (LSDs) lead to a progressive accumulation within the cell of macromolecular substrates that interfere with cellular function1. The disorders are caused by mutations in genes encoding a range of lysosomal enzymes, and the phenotype varies depending on the enzyme deficiency and the organs involved. Symptoms range from modest biochemical abnormalities to severe neurodegenerative disorders characterized by mental retardation, loss of motor and visual function, seizures and death.

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Figure 1: Retargeting of AAV vectors to the diseased brain endothelium.

Katie Vicari

References

  1. Platt, F.M. & Lachmann, R.H. Biochim. Biophys. Acta 1793, 737–745 (2009).

    Article  CAS  Google Scholar 

  2. Chen, Y.H., Chang, M. & Davidson, B.L. Nat. Med. 15, 1215–1218 (2009).

    Article  CAS  Google Scholar 

  3. Girod, A. et al. Nat. Med. 5, 1052–1056 (1999).

    Article  CAS  Google Scholar 

  4. Foust, K.D. et al. Nat. Biotechnol. 27, 59–65 (2009).

    Article  CAS  Google Scholar 

  5. Young, P.P., Fantz, C.R. & Sands, M.S. Exp. Neurol. 188, 104–114 (2004).

    Article  CAS  Google Scholar 

  6. Worgall, S. et al. Hum. Gene Ther. 19, 463–474 (2008).

    Article  CAS  Google Scholar 

  7. Cavazzana-Calvo, M. et al. Blood 112, abstact 821 (2008).

  8. Cearley, C.N. & Wolfe, J.H. J. Neurosci. 27, 9928–9940 (2007).

    Article  CAS  Google Scholar 

  9. Work, L.M. et al. Mol. Ther. 13, 683–693 (2006).

    Article  CAS  Google Scholar 

  10. Brooks, A.I. et al. Proc. Natl. Acad. Sci. USA 99, 6216–6221 (2002).

    Article  CAS  Google Scholar 

  11. Gao, G.P. et al. Proc. Natl. Acad. Sci. USA 99, 11854–11859 (2002).

    Article  CAS  Google Scholar 

  12. Schaffer, D.V., Koerber, J.T. & Lim, K.I. Annu. Rev. Biomed. Eng. 10, 169–194 (2008).

    Article  CAS  Google Scholar 

Download references

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Authors and Affiliations

  1. Federico Mingozzi and Katherine A. High are in the Division of Hematology, the Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.,

    Federico Mingozzi & Katherine A High

  2. Katherine A. High is also at the Howard Hughes Medical Institute, Philadelphia, Pennsylvania, USA.,

    Katherine A High

Authors
  1. Federico Mingozzi
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  2. Katherine A High
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Correspondence to Katherine A High.

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Mingozzi, F., High, K. Gateway to the diseased brain. Nat Med 15, 1123–1124 (2009). https://doi.org/10.1038/nm1009-1123

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