Biblio
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Treatment Paradigms for Retinal and Macular Diseases Using 3-D Retina Cultures Derived From Human Reporter Pluripotent Stem Cell Lines. Invest Ophthalmol Vis Sci. 2016;57(5):ORSFl1-ORSFl11.
Culture of pyramidal neural precursors, neural stem cells, and fibroblasts on various biomaterials. J Biomater Sci Polym Ed. 2018:1-38.
. 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 Res. 2019;37:101436.
. 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 Res. 2019;36:101408.
. 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.
. 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 Res. 2019;37:101435.
. 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 Res. 2019;37:101427.
. An induced pluripotent stem cell line (TRNDi010-C) from a patient carrying a homozygous p.R401X mutation in the NGLY1 gene. Stem Cell Res. 2019;39:101496.
Induced pluripotent stem cells for neural drug discovery. Drug Discov Today. 2019.
. Pharmacological analysis of CFTR variants of cystic fibrosis using stem cell-derived organoids. Drug Discov Today. 2019.
. Combined associations of a polygenic risk score and classical risk factors with breast cancer risk. J Natl Cancer Inst. 2020.
Four induced pluripotent stem cell lines (TRNDi021-C, TRNDi023-D, TRNDi024-D and TRNDi025-A) generated from fibroblasts of four healthy individuals. Stem Cell Res. 2020;49:102011.
. Generation and characterization of four Chediak-Higashi Syndrome (CHS) induced pluripotent stem cell (iPSC) lines. Stem Cell Res. 2020;47:101883.
. Disease modeling for Mucopolysaccharidosis type IIIB using patient derived induced pluripotent stem cells. Exp Cell Res. 2021:112785.
. Generation of an induced pluripotent stem cell line (TRNDi031-A) from a patient with Alagille syndrome type 1 carrying a heterozygous p. C312X (c. 936 T > A) mutation in JAGGED-1. Stem Cell Res. 2021;54:102447.
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.