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Review
. 2002 Apr 30;99(9):5760-2.
doi: 10.1073/pnas.102175599.

Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier

William S Sly  1 , Carole Vogler
Affiliations
Review

Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood- brain barrier

William S Sly et al. Proc Natl Acad Sci U S A. .
No abstract available

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Figures

Figure 1
Figure 1
An adult MPS VII (Sly Disease) mouse (Left) is much smaller than its phenotypically normal littermate (Right) and has facial dysmorphism with a broad shortened nose and short limbs.
See this image and copyright information in PMC

Comment on

References

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