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Loss of Extreme Long-Range Enhancers in Human Neural Crest Drives a Craniofacial Disorder.
Long HK, Osterwalder M, Welsh IC, Hansen K, Davies JOJ, Liu YE, Koska M, Adams AT, Aho R, Arora N, Ikeda K, Williams RM, Sauka-Spengler T, Porteus MH, Mohun T, Dickel DE, Swigut T, Hughes JR, Higgs DR, Visel A, Selleri L, Wysocka J. Long HK, et al. Among authors: porteus mh. Cell Stem Cell. 2020 Nov 5;27(5):765-783.e14. doi: 10.1016/j.stem.2020.09.001. Epub 2020 Sep 28. Cell Stem Cell. 2020. PMID: 32991838 Free PMC article.
Efficient scarless genome editing in human pluripotent stem cells.
Ikeda K, Uchida N, Nishimura T, White J, Martin RM, Nakauchi H, Sebastiano V, Weinberg KI, Porteus MH. Ikeda K, et al. Among authors: porteus mh. Nat Methods. 2018 Dec;15(12):1045-1047. doi: 10.1038/s41592-018-0212-y. Epub 2018 Nov 30. Nat Methods. 2018. PMID: 30504872
Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination.
Martin RM, Ikeda K, Cromer MK, Uchida N, Nishimura T, Romano R, Tong AJ, Lemgart VT, Camarena J, Pavel-Dinu M, Sindhu C, Wiebking V, Vaidyanathan S, Dever DP, Bak RO, Laustsen A, Lesch BJ, Jakobsen MR, Sebastiano V, Nakauchi H, Porteus MH. Martin RM, et al. Among authors: porteus mh. Cell Stem Cell. 2019 May 2;24(5):821-828.e5. doi: 10.1016/j.stem.2019.04.001. Cell Stem Cell. 2019. PMID: 31051134 Free article.
Neuronal defects in a human cellular model of 22q11.2 deletion syndrome.
Khan TA, Revah O, Gordon A, Yoon SJ, Krawisz AK, Goold C, Sun Y, Kim CH, Tian Y, Li MY, Schaepe JM, Ikeda K, Amin ND, Sakai N, Yazawa M, Kushan L, Nishino S, Porteus MH, Rapoport JL, Bernstein JA, O'Hara R, Bearden CE, Hallmayer JF, Huguenard JR, Geschwind DH, Dolmetsch RE, Paşca SP. Khan TA, et al. Among authors: porteus mh. Nat Med. 2020 Dec;26(12):1888-1898. doi: 10.1038/s41591-020-1043-9. Epub 2020 Sep 28. Nat Med. 2020. PMID: 32989314 Free PMC article.
High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia.
Vaidyanathan S, Salahudeen AA, Sellers ZM, Bravo DT, Choi SS, Batish A, Le W, Baik R, de la O S, Kaushik MP, Galper N, Lee CM, Teran CA, Yoo JH, Bao G, Chang EH, Patel ZM, Hwang PH, Wine JJ, Milla CE, Desai TJ, Nayak JV, Kuo CJ, Porteus MH. Vaidyanathan S, et al. Among authors: porteus mh. Cell Stem Cell. 2020 Feb 6;26(2):161-171.e4. doi: 10.1016/j.stem.2019.11.002. Epub 2019 Dec 12. Cell Stem Cell. 2020. PMID: 31839569 Free PMC article.
Stem Cell-Specific Mechanisms Ensure Genomic Fidelity within HSCs and upon Aging of HSCs.
Moehrle BM, Nattamai K, Brown A, Florian MC, Ryan M, Vogel M, Bliederhaeuser C, Soller K, Prows DR, Abdollahi A, Schleimer D, Walter D, Milsom MD, Stambrook P, Porteus M, Geiger H. Moehrle BM, et al. Cell Rep. 2015 Dec 22;13(11):2412-2424. doi: 10.1016/j.celrep.2015.11.030. Epub 2015 Dec 10. Cell Rep. 2015. PMID: 26686632 Free PMC article.
CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells.
Goodwin M, Lee E, Lakshmanan U, Shipp S, Froessl L, Barzaghi F, Passerini L, Narula M, Sheikali A, Lee CM, Bao G, Bauer CS, Miller HK, Garcia-Lloret M, Butte MJ, Bertaina A, Shah A, Pavel-Dinu M, Hendel A, Porteus M, Roncarolo MG, Bacchetta R. Goodwin M, et al. Sci Adv. 2020 May 6;6(19):eaaz0571. doi: 10.1126/sciadv.aaz0571. eCollection 2020 May. Sci Adv. 2020. PMID: 32494707 Free PMC article.
Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase.
Wilkes MC, Siva K, Chen J, Varetti G, Youn MY, Chae H, Ek F, Olsson R, Lundbäck T, Dever DP, Nishimura T, Narla A, Glader B, Nakauchi H, Porteus MH, Repellin CE, Gazda HT, Lin S, Serrano M, Flygare J, Sakamoto KM. Wilkes MC, et al. Among authors: porteus mh. Nat Commun. 2020 Jul 3;11(1):3344. doi: 10.1038/s41467-020-17100-z. Nat Commun. 2020. PMID: 32620751 Free PMC article.
181 results