Benetó, NoeliaCozar, MónicaGort i Mas, LauraPacheco, LauraVilageliu i Arqués, LluïsaGrinberg Vaisman, Daniel RaúlCanals Montferrer, Isaac2021-06-012021-06-012020-011873-5061https://hdl.handle.net/2445/177895Mutations in the NAGLU gene cause Sanfilippo B syndrome (mucopolysaccharidosis IIIB), a rare lysosomal storage disorder whose main symptom is a severe and progressive neurodegeneration for which no treatment is still available. Here, we generated two homozygous NAGLU-mutated cell lines using CRISPR/Cas9 editing in a healthy human induced pluripotent stem cell (hiPSC) line. These novel cell lines express pluripotency specific markers and maintain their capability to differentiate into all three germ layers in vitro while exhibit a normal karyotype. These mutated lines in combination with the isogenic control line will be useful to model in vitro Sanfilippo B syndrome.4 p.application/pdfengcc-by (c) Benetó, Noelia et al., 2019https://creativecommons.org/licenses/by/4.0/MucopolisacàridsMalalties neurodegenerativesMucopolysaccharidesNeurodegenerative Diseasesinfo:eu-repo/semantics/article6934182021-06-01info:eu-repo/semantics/openAccess