An iPSC-Derived Neuron Model of CLN3 Disease Facilitates Small Molecule Phenotypic Screening

Nihar Kinarivala, Ahmed Morsy, Ronak Patel, Angelica V. Carmona, Md Sanaullah Sajib, Snehal Raut, Constantinos M. Mikelis, Abraham Al-Ahmad, Paul C. Trippier

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14 Scopus citations

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Biochemistry, Genetics and Molecular Biology

Pharmacology, Toxicology and Pharmaceutical Science