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

38 Scopus citations

Fingerprint

Dive into the research topics of 'Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis'. Together they form a unique fingerprint.

Biochemistry, Genetics and Molecular Biology

Neuroscience