Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses

Overview of attention for article published in Orphanet Journal of Rare Diseases, April 2018

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Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease

Article in Advances in Cell and Gene Therapy (March 2024)

Adult-onset neuronal ceroid lipofuscinosis misdiagnosed as autoimmune encephalitis and normal-pressure hydrocephalus: a 10-year case report and literature review

Preprint in Research Square (February 2024)

Cellular Modeling of CLN6 with IPSC-derived Neurons and Glia

Preprint in bioRxiv (January 2024)

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