DOI: 10.1055/s-00029022

Journal of Pediatric Biochemistry

References

Worgall S, Sondhi D, Hackett NR , et al.
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

Hum Gene Ther 2008;
19 (5) 463-474

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