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Stem Cell Res. 2019 Oct 24;41:101616. doi: 10.1016/j.scr.2019.101616. [Epub ahead of print]

Generation of two compound heterozygous HGSNAT-mutated lines from healthy induced pluripotent stem cells using CRISPR/Cas9 to model Sanfilippo C syndrome.

Author information

1
Department Genetics, Microbiology and Statistics, Fac. Biology, University of Barcelona, CIBERER, IBUB, IRSJD, Spain.
2
Stem Cells, Aging and Neurodegeneration Group, Lund Stem Cell Center, University Hospital, Lund, Sweden. Electronic address: isaac.canals@med.lu.se.
PMID:
31731183
DOI:
10.1016/j.scr.2019.101616
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