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

[Image: see text] The neuronal ceroid lipofuscinoses (NCLs) are a family of rare lysosomal storage disorders. The most common form of NCL occurs in children harboring a mutation in the CLN3 gene. This form is lethal with no existing cure or treatment beyond symptomatic relief. The pathophysiology of...

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Pubblicato in:ACS Pharmacol Transl Sci
Autori principali: Kinarivala, Nihar, Morsy, Ahmed, Patel, Ronak, Carmona, Angelica V., Sajib, Md. Sanaullah, Raut, Snehal, Mikelis, Constantinos M., Al-Ahmad, Abraham, Trippier, Paul C.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Chemical Society 2020
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7551715/
https://ncbi.nlm.nih.gov/pubmed/33073192
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acsptsci.0c00077
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