Disease modeling for Mucopolysaccharidosis type IIIB using patient derived induced pluripotent stem cells. Exp Cell Res. 2021:112785..
Generation of two gene corrected human isogenic iPSC lines (NCATS-CL6104 and NCATS-CL6105) from a patient line (NCATS-CL6103) carrying a homozygous p.R401X mutation in the NGLY1 gene using CRISPR/Cas9. Stem Cell Res. 2021;56:102554.
Generation of an induced pluripotent stem cell line (TRNDi008-A) from a Hunter syndrome patient carrying a hemizygous 208insC mutation in the IDS gene. Stem Cell Res. 2019;37:101451..