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ArticleHuman gene therapy methods2012

Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.

Dolan Sondhi et al.PubMed ↗Full text ↗Publisher ↗

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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 23131032