Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

Marie S. Roberts, Shannon L. Macauley, Andrew M. Wong, Denis Yilmas, Sarah Hohm, Jonathan D. Cooper, Mark S. Sands

Research output: Contribution to journalArticlepeer-review

35 Scopus citations

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Medicine & Life Sciences