Generation of two compound heterozygous HGSNAT-mutated lines from healthy induced pluripotent stem cells using CRISPR/Cas9 to model Sanfilippo C syndrome.
Stem Cell Res. 2019 Dec;41:101616. doi: 10.1016/j.scr.2019.101616. Epub 2019 Oct 24.
Stem Cell Res. 2019.
PMID: 31731183
Free article.
No abstract available.