Biallelic correction of sickle cell disease-derived iPSCs confirmed at the protein level through serum-free iPS-sac/erythroid differentiation.Stem Cells Transl Med.(2020).
Generation of an induced pluripotent stem cell line (TRNDi004-I) from a Niemann-Pick disease type B patient carrying a heterozygous mutation of p.L43_A44delLA in the SMPD1 gene.Stem Cell Res37, 101436.(2019).
Generation of an induced pluripotent stem cell line (TRNDi005-A) from a Mucopolysaccharidosis Type IVA (MPS IVA) patient carrying compound heterozygous p.R61W and p.WT405del mutations in the GALNS gene.Stem Cell Res36, 101408.(2019).
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 Res37, 101451.(2019).
Generation of two induced pluripotent stem cell lines (NHLBIi001-A and NHLBIi001-B) from a healthy Caucasian female volunteer with normal cardiac function.Stem Cell Res41, 101627.(2019).
Generation of two tdTomato reporter induced pluripotent stem cell lines (NHLBIi003-A-1 and NHLBIi003-A-2) by AAVS1 safe harbor gene-editing.Stem Cell Res42, 101673.(2019).
A human induced pluripotent stem cell line (TRNDi007-B) from an infantile onset Pompe patient carrying p.R854X mutation in the GAA gene.Stem Cell Res37, 101435.(2019).
An induced pluripotent stem cell line (TRNDi006-A) from a MPS IIIB patient carrying homozygous mutation of p.Glu153Lys in the NAGLU gene.Stem Cell Res37, 101427.(2019).
An induced pluripotent stem cell line (TRNDi010-C) from a patient carrying a homozygous p.R401X mutation in the NGLY1 gene.Stem Cell Res39, 101496.(2019).
Heparin Promotes Cardiac Differentiation of Human Pluripotent Stem Cells in Chemically Defined Albumin-Free Medium, Enabling Consistent Manufacture of Cardiomyocytes.Stem Cells Transl Med.(2016).
Roles of H3K27me2 and H3K27me3 Examined during Fate Specification of Embryonic Stem Cells.Cell Rep17, 1369-1382.(2016).
Treatment Paradigms for Retinal and Macular Diseases Using 3-D Retina Cultures Derived From Human Reporter Pluripotent Stem Cell Lines.Invest Ophthalmol Vis Sci57, ORSFl1-ORSFl11.(2016).
Generation of GFP Reporter Human Induced Pluripotent Stem Cells Using AAVS1 Safe Harbor Transcription Activator-Like Effector Nuclease.Curr Protoc Stem Cell Biol29, 5A.7.1-5A.7.18.(2014).