Stemolecule™ CHIR99021

The aminopyrimidine CHIR99021 is the most selective inhibitor of glycogen synthase kinase 3 β (GSK-3β) reported to date^1,2. Unlike other potent inhibitors of GSK-3, CHIR99201 does not exhibit cross-reactivity against cyclin-dependent kinases (CDKs) and shows a 350-fold selectivity toward GSK-3β compared to CDKs³. Along with the elimination of differentiation-inducing signaling from mitogen-activated protein kinases, using CHIR99021 to block the activity of GSK-3β enables the self-renewal of embryonic stem cells⁴.

Specifications

Product Name: Stemolecule CHIR99021

Catalog Number: 04-0004

Size

• 2 mg (Cat. No. 04-0004)
• 10 mg (Cat. No. 04-0004-10)
• 2 mg in 10 mM DMSO solution (Cat. No. 04-0004-02)

Alternate Name(s): 6-[[2-[[4-(2,4-dichlorophenyl)-5-(5-methyl-1H-imidazol-2-yl)-2 pyrimidinyl]amino]ethyl]amino]-3-pyridinecarbonitrile

Chemical Formula: C₂₂H₁₈Cl₂N₈

Molecular Weight: 465.34

CAS Number: 252917-06-9

Purity: Greater than 95 % by HPLC analysis

Formulation: Off-white solid

Solubility: Reconstitute in DMSO to the desired concentration. For reconstitution instructions please reference product specifications sheet.

Storage and Stability: Store powder at 4 °C protected from light. Information about the stability of Stemolecules in solution is largely not available. As a general guideline, we recommend that stock solution be freshly made and stored in aliquots at −20 °C. The effect of storage of stock solutions should be verified for each application.

Quality Control: The purity of CHIR99021 was determined by HPLC analysis. The accurate mass was determined by mass spectrometry. Cellular toxicity of CHIR99021 was tested on mouse embryonic stem cells.

Documents

Specification Sheets:

• 04-0004 Specifications Sheet
• 04-0004-10 Specifications Sheet
• 04-0004-02 Specifications Sheet

Safety Data Sheets:

• 04-0004 and 04-0004-10 Safety Data Sheet
• 04-0004-02 Safety Data Sheet

References

1. Sato, N., Meijer, L., Skaltsounis, L., Greengard, P., and Brivanlou, A.H. Maintenance of pluripotency in human and mouse embryonic stem cells through activation of Wnt signaling by a pharmacological GSK-3-specific inhibitor. Nat Med 10: 55-63 (2004).
2. Besser, D. (2004) Expression of nodal, lefty-a, and lefty-B in undifferentiated human embryonic stem cells requires activation of Smad 2/3. J Biol Chem 279: 45076-45084 (2004).
3. Ying, Q.L., Wray, J., Nichols, J., Batlle-Morera, L., Doble, B., Woodgett, J., Cohen, P., and Smith, A. The ground state of embryonic stem cell self renewal. Nature 453: 519-523 (2008).
4. Polychronopoulos, P., Magiatis, P., Skaltsounis, A.L., Myrianthopoulos, V., Mikros, E., Tarricone, A., Musacchio, A., Roe, S.M., Pearl, L., Leost, M., Greengard, P., and Meijer, L. Structural basis for the synthesis of indirubins as potent and selective inhibitors of glycogen synthase kinase-3 and cyclin-dependent kinases. J Med Chem 47: 935-946 (2004).

Additional Publications

• Lowe MG; Yen M-R; Hsu F-M; Hoshhama L; Hu Z; Chitiashvili T; Hunt TJ; Gorgy I; Bernard M; Wanaitha SE; Chen P-Y; Clark AT. EED is required for mouse primordial germ cell differentiation in the embryonic gonad. Developmental Cell 57:1482.e5 (2022).
• SIM EZ; Enomoto T; Shiraki N; Furuta N; Kashio S; Kambe T; Tsuyama T; Arakawa A; Ozawa H; Yokoyama M; Miuri M; Kume S. Methionine metabolism regulates pluripotent stem cell pluripotency and differentiation through zinc mobilization. Cell Rep 40:111120 (2022).
• Liang L ;Tian Y; Feng L; Wang C; Feng G; Stacey GN; Shyh-Change N; Wu J; Hu B; Li W: Hao J; Wang L; Wang Y. Single-cell transcriptomics reveals the cell fate transitions of human dopaminergic progenitors derived from hESCs. Stem Cell Res Therap 13:412 (2022).
• Lieberman R; Cortes LK; Gao G; Park H; Wang B; Jones PL; Hunter RB; Leonard JP; Barker RH. Human iPSC-derived astrocytes generated from donors with globoid cell leukodystrophy display phenotypes associated with disease. PLOS One 17:e0271360 (2022).
• Wang T; Liu H; Itoh K; Oh S; Zhao L ;Murata D; Sesaki H; Hartung T; Na CH; Wang J. C9orf72 regulates energy homeostasis by stabilizing mitochondrial complex I assembly. Cell Metabolism 33:531-546.e9 (2021).
• Facoioli R; Lojudice FH; Anauate AC; Maquigussa E; Nishiura JL; Heilberg IP; Sogayar MC; Boir MA. Kidney organoids generated from erythroid progenitors cells of patients with autosomal dominant polycystic kidney disease. PLOS One 16:e0252156 (2021).
• Yang D; Patel S; Szlachcic J; Scaduto S; Putluri N; Sreekumar A; Suliburk J; Metzker M; Balasubramanyam A; Borowiak M. Pancreatic Differentiation of Stem Cells Reveals Pathogenesis of a Syndrome of Ketosis-Prone Diabetes. Diabetes 70:2419 (2021).
• Habekost M; Qvist P; Denham M; Holm IE; Jørgensen AL. Directly Reprogrammed Neurons Express MAPT and APP Splice Variants Pertinent to Ageing and Neurodegeneration. Molecular Neurobiol 176:1 (2021).
• Tan JJ; Guyette JP; Miki K; Xiao L Kaur G; Wu T; Zhu L; Hansen KJ; Ling K-H; Milan DJ; Ott HC. Human iPS-derived pre-epicardial cells direct cardiomyocyte aggregation expansion and organization in vitro. Nature Commun 12:4997 (2021).
• Chapkin RS; Davidson LA; Park H; Jin U-H; Fan Y-Y; Cheng Y; Hensel ME; Landrock KK; Allred C; Menon R; Klemashevich C; Jayaraman A; Safe S. Role of the aryl hydrocarbon teceptor (AhR) in mediating the effects of coffee in the colon. Mol Nutrition Food Res 65:2100539 (2021).
• Huang X; Park K; Gontarz P Zhang B; Pan J; McKenzie Z; Fischer LA; Dong C; Dietmann S; Xing X; Shliaha PB; Yang J; Li D ;Ding J; Lungjangwa T; Mitalipova M; Khan S; Imsoonthornruksa S; Jensen M; Wang T; Kadoch C; Raenisch R; Wang J; Theunissen TW. OCT4 cooperates with distinct ATP-dependent chromatin remodelers in naïve and primed pluripotent states in human. Nature Commun 12:5123 (2021).
• He X; Smith SE; Chen S; Lil H; Wu D; Meneses-Giles PI; Wang Y; Hembree M; Yi K; Zhao X; Guo F; Unruh JR; Maddera LE; Ye Z; Scott A; Perera A; Wang Y; Zhao C; Bae KM; Box A; Haug JS; Tao F; Hu D; Hansen DM; Qian P; Saha S; Dixon D; Anant S; Zhang D; Lin EH; Sun W; Wiedemann LM; Li L;. Tumor-initiating stem cell shapes its microenvironment into an immunosuppressive barrier and pro-tumorigenic niche. Cell Rep 36:109674 (2021).
• Ulutas MS. Interaction of intestinal microbiota and intestinal epithelial health in high carbohydrate diet. Ph.D. Theseis, Middle East Technical Univ : (2021).
• Mao Z; Li Y; Huang L; Chen Y; Luo H; Zhang S; Chen H; . Generation of an induced pluripotent stem cell line HUSTTJi001-A from a Moyamoya disease patient with RNF213 gene mutation. Stem Cell Res 57:102575 (2021).
• Gabriel V; Zdyrski C; Sahoo DK; Dao K; Mourgois-Mochel A; Atherly T; Martinez MN; Volpe DA; Kopper J; Allenspach K; Mochel JP. Canine Intestinal Organoids in a Dual-Chamber Permeable Support System2022. J Vis Exp 181:e63612 (2022).
• Yeo G; xiang J; Mueller J; Luo E-C; Yee B; Schafer D; Schmok J; Tan F; Rothamel K; McVicar R; Kwong E; Croker B; Jones K; Her H-L; Chen C-Y; Vu A; Jin W; Park S; Le P; Brennan K; Kofman E; Li Y; Tankka A; Dong K; Song Y; Clark A; Carlin A; Van Nostrand E; Leibel S. Discovery and functional interrogation of SARS-CoV2 protein-RNA interactions. Research Square :doi.org/10.21203/rs.3.rs-1394331/v1 (2022).
• inamura S; Yoshimoto K; Terada S; Takamuro K; Kamei K-i. In vitro culture at 39 °C during hepatic maturation of human ES cells facilitates hepatocyte-like cell functions. Sci Reports 12:5515 (2022).
• Kase Y; Sato T; Okano Y; Okano H. The GADD45G/p38 MAPK/CDC25B signaling pathway enhances neurite outgrowth by promoting microtubule polymerization. iScience 25:104089 (2022).
• Li Z; Lui KN-C; Lau S-T; Lai FP-L; Li P; Chung P; Wong K; Tam P; Cracia-Barcelo M-M; Hui C-c; Sham P; Ngan E. Hedgehog-induced-oxidative-phosphorylation rescues neuronal differentiation defect of human enteric neural crest cells underlying Hirschsprung disease. Research Square :doi.org/10.21203/rs.3.rs-1433241/v1 (2022).
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• Hageb A; Thalheim T: Nattamai KJ; Möhrle B; Saçma M; Sakk V; Thielecke L; Cornils K; Grandy C; Port F; Gottschalk K-E; Mallm J-P; Glauche I; Galle J; Mulaw MA; Geiger H. Reduced adhesion of aged intestinal stem cells contributes to an accelerated clonal drift. Life Science Alliance 5:DOI: 10.26508/lsa.202201408 (2022).
• Wen J; Song J; Bai Y; Liu T; Cai X; Mei L ;Ma L ; He C; Feng Y. A Model of Waardenburg Syndrome Using Patient-Derived iPSCs With a SOX10 Mutation Displays Compromised Maturation and Function of the Neural Crest That Involves Inner Ear Development. Front Cell Dev Biol 9:720858 (2022).
• Mou X; Shah J; Bhattacharya R; Kalejaiye TD; Sun B: Hsu P-C; Musah S. A Biomimetic Electrospun Membrane Supports the Differentiation and Maturation of Kidney Epithelium from Human Stem Cells. Bioengineering 9:188 (2022).
• Wange W; Su L; Wang Y; Li C; Ji F; Jiao J. Endothelial Cells Mediated by UCP2 Control the Neurogenic-to-Astrogenic Neural Stem Cells Fate Switch During Brain Development. Advanced Sci 9:2105208 (2022).
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• Teleahmad S' Salari A; Samadian A' Chee SH' Hwang D' Lee B'; Bayarsaikjan D; Bayarsaikjan G; Lee J; Park JH; Hassani S-N; Baharvand H; Salekdeh GH. BMP4 signaling plays critical roles in self-renewal of R2i mouse embryonic stem cells. Biochem Biophys Res Commun 617:1-8 (2022).
• Dong C; Fu S; Karvas RM; Chew B; Fischer LA; Xing X; harrison JK; popli P; Kommagani R; Wang T; Zhang B; Theunissen TW. A genome-wide CRISPR-Cas9 knockout screen identifies essential and growth-restricting genes in human trophoblast stem cells. Nature Commun 13:2548 (2022).
• Samanta MK; gayen S; Harris C; Maclary E; Murata-Nakamura Y; Malcore RM; Porter TS; Garay PM; Vallianatos CN; Samollow PB; Iwase S; Kalantry S. Activation of Xist by an evolutionarily conserved function of KDM5C demethylase. Nature Commun 13:2602 (2022).
• Li H; Jiang H; Li L; Yan Z; Feng J. Generation of human A9 dopaminergic pacemakers from induced pluripotent stem cells. Molec Phychiatry 44682:DOI: 10.1038/s41380-022-01628-1 (2022).
• Kremer V; Stanicek : van Ingen E; Bink DI; Hilderink S; Tijsen AJ; Wittig I; Mägdefessel L; Nossent Ay; Boon RA. Long non-coding RNA MEG8 induces endothelial barrier through regulation of microRNA-370 and -494 processing . J Cell Sci in press: (2022).
• Yoo J; La Y; Lee EJ; Choi J-J; Oh J; Thang NX; Hong K. ATP-Dependent chromatin remodeler CHD9 controls the proliferation of embryonic stem cells in a cell culture condition-dependent manner. Biology 9:428 (2020).
• Liang AS; Pagano JE; Chrzan CA; McKinnon RD. Suicide transport blockade of motor neuron survival generates a focal graded injury and functional deficit. Neural Regen Res 16:1281-1287 (2021).
• Ben-Reuven L; Reiner O. Toward Spatial Identities in Human Brain Organoids-on-Chip Induced by Morphogen-Soaked Beads. Bioengineering 7:164 (2020).
• Pereiro X; Miltner AM; La Torre A; Vecino E. Effects of Adult Müller Cells and Their Conditioned Media on the Survival of Stem Cell-Derived Retinal Ganglion Cells. Cells 9:1759 (2020).
• Shi G. Ddx56 maintains proliferation of mouse embryonic stem cells via ribosome assembly and interaction with the Oct4/Sox2 complex. Stem Cell Res Therap 301:314 (2020).
• Wang S-H; Wang X-P. Generation of an induced pluripotent stem cell (iPSC) line (THSJTUi001-A) from a Wilson's disease patient harboring a homozygous Arg778Leu mutation in ATP7B gene. Stem Cell Res 49:102050 (2020).
• Wang T; Li J; Xiao Y; Fu B; Wang P; Bai X; Cai G; Chen X; Li Q. Generation of induced pluripotent stem cell PLAFMCi002-A derived from peripheral blood mononuclear cells of polycystic kidney disease patient with PKD1 mutation. Stem Cell Res 49:102039 (2020).
• Park M; Kwon J; Shin H-J; Moon SM; Kim SB; Shin US; Han Y-H; Kim Y. Butyrate enhances the efficacy of radiotherapy via FOXO3A in colorectal cancer patient‑derived organoids. Int J Oncol 57:1307-1318 (2020).
• Fernandes HJR; Patikas M; Foskolou S; Field SF; Park J-E; Myrne ML; Bassett AR; Metzakopian E. Single-Cell Transcriptomics of Parkinson’s Disease Human In Vitro Models Reveals Dopamine Neuron-Specific Stress Responses. Cell Reports 33:108263 (2020).
• Kim M; Hwang S-U; Yoon JD; Jeong YW; Kim E; Hyun S-H. Optimized Approaches for the Induction of Putative Canine Induced Pluripotent Stem Cells from Old Fibroblasts Using Synthetic RNAs. Animals 10:1848 (2020).
• Branca MA; Dias TP Cotovio JP; Rodrigues CAV; Fernandes TG; Cabra; JMS; Diogo MM. 3D Microwell Platform for Cardiomyocyte Differentiation of Human Pluripotent Stem Cells. Methods Mol Biol doi.org/10.1007/7651_2020_336: (2020).
• Habekost M; Jørgensen AL; Qvist P; Denham M. MicroRNAs and Ascl1 facilitate direct conversion of porcine fibroblasts into induced neurons. Stem Cell Res 48:101984 (2020).
• von Troyer M. Establishment of an alginate based 3D culture system for the generation of dopaminergic neurons. Masters Thesis, Univ Innsbruck : (2020).
• Kase Y; Okano H. Expression of ACE2 and a viral virulence-regulating factor CCN family member 1 in human iPSC-derived neural cells: implications for COVID-19-related CNS disorders. Inflammat Regen 40:32 (2020).
• Dignum T; Vamum-Finney B; Dozono S; Hadland B. Generation of AGM-derived Akt-EC (AGM-EC). Research Square :doi.org/10.21203/rs.3.pex-986/v1 (2020).
• Sato T; Higashioka K; Kakurai H; Yamamoto T; Goshima N; Ueno M; Sotozono C. Core Transcription Factors Promote Induction of PAX3-Positive Skeletal Muscle Stem Cells. Stem Cell Rep 13:352-365 (2019).
• Narcisi R; Koevoet WJLM; van Osch GJVM. Expansion and Chondrogenic Differentiation of Human Bone Marrow-Derived Mesenchymal Stromal Cells. Osteoporosis Osteoarthritis 2221:15-28 (2020).
• Shan H; Ye J; Zhuang T; TIanRui H; HaiPing X; ShengSheng Z; XinYue H; HanYu L; Lun W; ShuYang Z. Establishment of an induced pluripotent stem cell line PUMCHi004-A from a hereditary transthyretin amyloid cardiomyopathy patient with transthyretin (TTR) p.Asp38Asn mutation. Stem Cell Res :102022 (2020).
• Shen H; Ye J; Ping XH; Yue HX; Yang ZS. Establishment of an induced pluripotent stem cell line from a patient with hereditary transthyretin amyloidosis carrying transthyretin (TTR) mutation p.Phe53Val. Stem Cell Res https://doi.org/10.1016/j.scr.2020.101940: (2020).
• Hiller BM; Marmion DJ; Gross RM; Thomson CA; Chavez CA; Brundin P; Wakeman DR; McMahon CW; Kordower JH. Mitomycin-C treatment during differentiation of induced pluripotent stem cell-derived dopamine neurons reduces proliferation without compromising survival or function in vivo. Stem Cells Translational Medicine :DOI: 10.1002/sctm.20-0014 (2020).
• Chen M; Miamaitili M; Habekost M; Gill KP; Mermet-Joret N; Nabavi S; Febbbraro F; Denham M. Rapid generation of regionally specified CNS neurons by sequential patterning and conversion of human induced pluripotent stem cells. Stem Cell Res :https://doi.org/10.1016/j.scr.2020.101945 (2020).
• Li CH; Coffey EL; Dall'Agnese A; Hannett NM; Tang X; Henninger JE; Platt JM; Oksuz O; Zamudio AV; Afeyan LK; Schuijers J; Liu XS; Markoulaki S; Lungjangwa T; LeRoy G; Svoboda DS; Wogram E; Lee TI; Jaenisch R; Young RA. MeCP2 links heterochromatin condensates and neurodevelopmental disease. Nature :doi.org/10.1038/s41586-020-2574-4 (2020).
• Scott A; Olack B; Rouch JD; Khalil HA; Kokubun BA; Lei NY; Wang J; Solorzano S; Lewis M; Dunn JCY; Stelzner MG; Niland JC; Martin MG. Comparison of Surgical and Cadaveric Intestine as a Source of Crypt Culture in Humans. Cell Transplantation 29:1-29 (2020).
• Gao X; Li T; Cahan P; Zhao Y; Yourick JJ; Sprando RL. Hepatocyte-like cells derived from human induced pluripotent stem cells using small molecules: implications of a transcriptomic study. Stem Cell Res Therap 11:393 (2020).
• Kidwai F; Mui BWH; Arora D; Iqbal K; Hockaday M; Fernandez de Castro Diaz L; Cherman N; Martin D; Myneni VD; Ahmad M; Futrega K; Ali S; Merling RK; Karfman DS; Lee J; Roberry PG. Lineage-specific differentiation of osteogenic progenitors from pluripotent stem cells reveals the FGF1-RUNX2 association in neural crest-derived osteoprogenitors. Stem Cells :doi.org/10.1002/stem.3206 (2020).
• Zaborski Silva IZ; Waloski Robert A; Cabrera Cabo G; SPangenberg L; Stimamiglio MA; Dallagiovanna B; Gradia DF; Shigunov P. Effects of PUMILIO1 and PUMILIO2 knockdown on cardiomyogenic differentiation of human embryonic stem cells culture. PLoS ONE 15:e0222373 (2020).
• Long P; Liu Z; Wu B; Chen J; Sun C; Wang F; Huang Y; Chen H; Li Q; Ma Y. Generation of an induced pluripotent stem cell line from chorionic villi of a Patau syndrome spontaneous abortion. Stem Cell Res 45:101789 (2020).
• Ambrosini YM; Park Y; Jergens AE; Shin W; Min S; Atherly T; Borcherding DC; Jang J; Allenspach K; Mochel JP; Kim HJ. Recapitulation of the accessible interface of biopsy-derived canine intestinal organoids to study epithelial-luminal interactions. PLoS ONE 15:e231423 (2020).
• Liu Y; Cheng J; Siejka-Zelińska P; Weldon C; Roberts H; Lopopolo M; Magri A; D'Arienzo V; Harris JM; McKeating JA; Song C-X. Accurate targeted long-read DNA methylation and hydroxymethylation sequencing with TAPS. Genome Biol 21:54 (2020).
• Altay G; Batlle E; Fernàndez-Majada V; Martinez E. In vitro self-organized mouse small intestinal epithelial monolayer protocol. Bio-protocol 10:e2514 (2020).
• Ahfeldt T; Ordureau A; Bell C; Sarrafha L; Sun C; Piccinotti S; Crass T; Parfitt GM; Paulo JA; Yanagawa J; Uozumi T; Kiyota Y; Harper JW' Rubin LL. Pathogenic Pathways in Early-Onset Autosomal Recessive Parkinson's Disease Discovered Using Isogenic Human Dopaminergic Neurons. Stem Cell Rep in press:doi.org/10.1016/j.scemcr.2019.12.005 (2020).
• Chen D; Sun N; Hou L; Kim R; Faith J; Aslanyan M; Tao Y; Zheng Y; Fu J; Liu W; Kellis M; Clark A. Human Primordial Germ Cells Are Specified from Lineage-Primed Progenitors. Cell Rep 29:4568-4582.e5 (2019).
• Iannielli A; Ugonlini GS; Cordiglieri C; Bido S; Rubio A; Colasante G; Valtorta M; Cabassi T; Rasponi M; Broccoli V. Reconstitution of the Human Nigro-striatal Pathway on-a-Chip Reveals OPA1-Dependent Mitochondrial Defects and Loss of Dopaminergic Synapses. Cell Rep 29:4646-4656.e4 (2019).
• Fujumichi Y; Otsuka K; Tomita M; Iwasaki T. An Efficient Intestinal Organoid System of Direct Sorting to Evaluate Stem Cell Competition in Vitro. Sci Rep 9:20297 (2019).
• Mahajani S; Raina A; Fokken C; Kügler S; Bähr M. Homogenous generation of dopaminergic neurons from multiple hiPSC lines by transient expression of transcription factors. Cell Death Disease 10:898 (2019).
• Vahdat S; Pahlavan S' Mahmoudi E; Barekat M; Ansari H; Bakhshandeh B; Aghdami N; Baharvand H. Expansion of Human Pluripotent Stem Cell-derived Early Cardiovascular Progenitor Cells by a Cocktail of Signaling Factors. Sci Rep 9:16006 (2019).
• Saraiva JE. Population-Level Dynamics of Human Pluripotent Stem Cell Fate Transitions. Universidade de Lisboa Ph.D. Thesis: (2019).
• Gantner CWB. Investigating Brain Repair and Development Using Stem Cells. Ph. D. Thesis, University of Melbourne : (2019).
• Koutmani Y; Gampierakis IA; Polissidis A; Ximerakis M: Koutsoudaki PN; Polyzos A; Agrogiannis G; Karaliota S; Thomaidou D; Rubin LL; Politis PK; Karakis KP. CRH Promotes the Neurogenic Activity of Neural Stem Cells in the Adult Hippocampus. Stem Cell Rep 28:932-945.e7 (2019).
• Leitner D; Ramamoorthy M; Dejosez M; Zwaka TP. Immature mDA Neurons Ameliorate Motor Deficits in a 6-OHDA Parkinson's Disease Mouse Model and are Functional after Cryopreservation. Stem Cell Res in press:doi.org/10.1016/j.scr.2019.101617 (2019).
• Atmanli A; Hu D; Deiman FE; van de Vrugt AM; Black III LD; Domian IJ. Multiplex Live Single-Cell Transcriptional Analysis Demarcates Cellular Functional Heterogeneity. eLIFE 8:e49599 (2019).
• Komura S; Ito K; Ohta S; Ukai T; Kabatga M; Itakura F; Semi K; Matsuda Y; Hashimoto K; Shibatga H; Sone M; Jo N; Sekiguchi K; Ohno T; Akiyama H; Shimizu K ;Woltjen K; Ozawa M; Toguchida J; Yamamoto T: Yamada Y. Cell-type dependent enhancer binding of the EWS/ATF1 fusion gene in clear cell sarcomas. Nature Commun 10:3999 (2019).
• Leibel SL; Winquist A; Tseu I; Wang J; Luo D; Shojaie S; Nathan N; Snyder E; Post M. Reversal of Surfactant Protein B Deficiency in Patient Specific Human Induced Pluripotent Stem Cell Derived Lung Organoids by Gene Therapy. Sci Rep 9:13450 (2019).
• LeRoy G; Oksuz O; Descostes D; Aoi Y; Ganai RA; Ortabozkoyun Kara H; Yu R-R; Lee C-H; Stafford J; Shilatifard A; Reinberg D. LEDGF and HDGF2 relieve the nucleosome-induced barrier to transcription in differentiated cells. Sci Advances 5:eaay3068 (2019).
• Durens M; Nestor J; Herold K Niescier RF; Lunden FW; Phillips AW; Lin Y-C; Nestor MW. High-content interrogation of human induced pluripotent stem cell-derived cortical organoid platforms. bioRxiv http://dx.doi.org/10.1101/697623. : (2019).
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• Li J-J; Lin X; Tang C; Lu Y-Q; Hu X; Zuo E; Li H; Ying W; Sun Y; Lai L-L; Chen H-Z; Guo X0X; Zhang Q-J; Wu S; Zhou C; Shen X; Wang Q: Lin T-T; Ma L-X; Wang N; Krainer AR; Shi L Yang H; Chen W-J. Disruption of splicing-regulatory elements using CRISPR/Cas9 rescues spinal muscular atrophy in human iPSCs and mice. National Sci Rev in press:nwz131 (2019).
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