Generation of two NAGLU-mutated homozygous cell lines from healthy induced pluripotent stem cells using CRISPR/Cas9 to model Sanfilippo B syndrome

Stem Cell Res. 2020 Jan:42:101668. doi: 10.1016/j.scr.2019.101668. Epub 2019 Nov 29.

Authors

Noelia Benetó¹, Monica Cozar¹, Laura Gort², Laura Pacheco², Lluïsa Vilageliu¹, Daniel Grinberg¹, Isaac Canals³

Affiliations

¹ Department of Genetics, Microbiology and Statistics, Fac. Biology, University of Barcelona, CIBERER, IBUB, IRSJD, Spain.
² Secció d'Errors Congènits del Metabolisme -IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic, IDIBAPS, CIBERER, Barcelona, Spain.
³ Stem Cells, Aging and Neurodegeneration Group, Lund Stem Cell Center, University Hospital, Lund, Sweden.

PMID: 31825816
DOI: 10.1016/j.scr.2019.101668

No abstract available

MeSH terms

CRISPR-Cas Systems / genetics*
Cell Line
Homozygote
Humans
Induced Pluripotent Stem Cells / metabolism*
Mucopolysaccharidosis III / genetics*
Mucopolysaccharidosis III / pathology