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Items: 1 to 50 of 130

1.

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.

Cell Stem Cell. 2019 Dec 11. pii: S1934-5909(19)30457-6. doi: 10.1016/j.stem.2019.11.002. [Epub ahead of print]

PMID:
31839569
2.

Functional significance of U2AF1 S34F mutations in lung adenocarcinomas.

Esfahani MS, Lee LJ, Jeon YJ, Flynn RA, Stehr H, Hui AB, Ishisoko N, Kildebeck E, Newman AM, Bratman SV, Porteus MH, Chang HY, Alizadeh AA, Diehn M.

Nat Commun. 2019 Dec 13;10(1):5712. doi: 10.1038/s41467-019-13392-y.

3.

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.

Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH.

Nat Commun. 2019 Dec 4;10(1):5624. doi: 10.1038/s41467-019-13620-5.

4.

Reply to "Efficient Nuclease-free HR by Clade F AAV Requires High MOIs with High Quality Vectors".

Dudek AM, Porteus MH.

Mol Ther. 2019 Dec 4;27(12):2063. doi: 10.1016/j.ymthe.2019.11.004. Epub 2019 Nov 11. No abstract available.

PMID:
31735604
5.

High-efficiency CRISPR induction of t(9;11) chromosomal translocations and acute leukemias in human blood stem cells.

Jeong J, Jager A, Domizi P, Pavel-Dinu M, Gojenola L, Iwasaki M, Wei MC, Pan F, Zehnder JL, Porteus MH, Davis KL, Cleary ML.

Blood Adv. 2019 Oct 8;3(19):2825-2835. doi: 10.1182/bloodadvances.2019000450.

6.

AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs.

Dudek AM, Porteus MH.

Mol Ther. 2019 Oct 2;27(10):1701-1705. doi: 10.1016/j.ymthe.2019.09.005. Epub 2019 Sep 12. No abstract available.

PMID:
31537456
7.

Early Tacrolimus Concentrations After Lung Transplant Are Predicted by Combined Clinical and Genetic Factors and Associated With Acute Kidney Injury.

Miano TA, Flesch JD, Feng R, Forker CM, Brown M, Oyster M, Kalman L, Rushefski M, Cantu E 3rd, Porteus M, Yang W, Localio AR, Diamond JM, Christie JD, Shashaty MGS.

Clin Pharmacol Ther. 2020 Feb;107(2):462-470. doi: 10.1002/cpt.1629. Epub 2019 Oct 20.

PMID:
31513279
8.

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I.

Gomez-Ospina N, Scharenberg SG, Mostrel N, Bak RO, Mantri S, Quadros RM, Gurumurthy CB, Lee C, Bao G, Suarez CJ, Khan S, Sawamoto K, Tomatsu S, Raj N, Attardi LD, Aurelian L, Porteus MH.

Nat Commun. 2019 Sep 6;10(1):4045. doi: 10.1038/s41467-019-11962-8.

9.

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.

Park SH, Lee CM, Dever DP, Davis TH, Camarena J, Srifa W, Zhang Y, Paikari A, Chang AK, Porteus MH, Sheehan VA, Bao G.

Nucleic Acids Res. 2019 Sep 5;47(15):7955-7972. doi: 10.1093/nar/gkz475.

10.

CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells.

Dever DP, Scharenberg SG, Camarena J, Kildebeck EJ, Clark JT, Martin RM, Bak RO, Tang Y, Dohse M, Birgmeier JA, Jagadeesh KA, Bejerano G, Tsukamoto A, Gomez-Ospina N, Uchida N, Porteus MH.

iScience. 2019 May 31;15:524-535. doi: 10.1016/j.isci.2019.04.036. Epub 2019 May 4.

11.

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.

Cell Stem Cell. 2019 May 2;24(5):821-828.e5. doi: 10.1016/j.stem.2019.04.001.

PMID:
31051134
12.

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.

Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH.

Nat Commun. 2019 Apr 26;10(1):2021. doi: 10.1038/s41467-019-10080-9.

13.

Gene correction for SCID-X1 in long-term hematopoietic stem cells.

Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH.

Nat Commun. 2019 Apr 9;10(1):1634. doi: 10.1038/s41467-019-09614-y. Erratum in: Nat Commun. 2019 Apr 26;10(1):2021. Nat Commun. 2019 Dec 4;10(1):5624.

14.

A New Class of Medicines through DNA Editing.

Porteus MH.

N Engl J Med. 2019 Mar 7;380(10):947-959. doi: 10.1056/NEJMra1800729. Review. No abstract available.

PMID:
30855744
15.

Identification of preexisting adaptive immunity to Cas9 proteins in humans.

Charlesworth CT, Deshpande PS, Dever DP, Camarena J, Lemgart VT, Cromer MK, Vakulskas CA, Collingwood MA, Zhang L, Bode NM, Behlke MA, Dejene B, Cieniewicz B, Romano R, Lesch BJ, Gomez-Ospina N, Mantri S, Pavel-Dinu M, Weinberg KI, Porteus MH.

Nat Med. 2019 Feb;25(2):249-254. doi: 10.1038/s41591-018-0326-x. Epub 2019 Jan 28.

PMID:
30692695
16.

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.

Nat Methods. 2018 Dec;15(12):1045-1047. doi: 10.1038/s41592-018-0212-y. Epub 2018 Nov 30.

PMID:
30504872
17.

Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6.

Bak RO, Dever DP, Reinisch A, Cruz Hernandez D, Majeti R, Porteus MH.

Elife. 2018 Nov 16;7. pii: e43690. doi: 10.7554/eLife.43690.

18.

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.

Lattanzi A, Meneghini V, Pavani G, Amor F, Ramadier S, Felix T, Antoniani C, Masson C, Alibeu O, Lee C, Porteus MH, Bao G, Amendola M, Mavilio F, Miccio A.

Mol Ther. 2019 Jan 2;27(1):137-150. doi: 10.1016/j.ymthe.2018.10.008. Epub 2018 Oct 17.

19.

Protect NIH's DNA advisory committee.

Adelman ZN, Albritton LM, Boris-Lawrie K, Buchmeier MJ, Cannon P, Cho M, DiGiusto D, Donahue JK, Federoff HJ, Hammarskjold ML, Hardison AD, Hearing P, Lee B, Lee DA, Porteus MH, Ross LF, Ross SR, Wooley DP, Zoloth L.

Science. 2018 Oct 26;362(6413):409-410. doi: 10.1126/science.aav2483. No abstract available.

PMID:
30361364
20.

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting.

Charlesworth CT, Camarena J, Cromer MK, Vaidyanathan S, Bak RO, Carte JM, Potter J, Dever DP, Porteus MH.

Mol Ther Nucleic Acids. 2018 Sep 7;12:89-104. doi: 10.1016/j.omtn.2018.04.017. Epub 2018 May 3.

21.

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification.

Vaidyanathan S, Azizian KT, Haque AKMA, Henderson JM, Hendel A, Shore S, Antony JS, Hogrefe RI, Kormann MSD, Porteus MH, McCaffrey AP.

Mol Ther Nucleic Acids. 2018 Sep 7;12:530-542. doi: 10.1016/j.omtn.2018.06.010. Epub 2018 Jun 30.

22.

Interferon priming is essential for human CD34+ cell-derived plasmacytoid dendritic cell maturation and function.

Laustsen A, Bak RO, Krapp C, Kjær L, Egedahl JH, Petersen CC, Pillai S, Tang HQ, Uldbjerg N, Porteus M, Roan NR, Nyegaard M, Denton PW, Jakobsen MR.

Nat Commun. 2018 Aug 30;9(1):3525. doi: 10.1038/s41467-018-05816-y.

23.

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.

Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai SY, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD.

Blood. 2018 Oct 25;132(17):1737-1749. doi: 10.1182/blood-2018-03-840702. Epub 2018 Aug 28.

24.

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.

Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S, Camarena J, Lee CM, Park SH, Wiebking V, Bak RO, Gomez-Ospina N, Pavel-Dinu M, Sun W, Bao G, Porteus MH, Behlke MA.

Nat Med. 2018 Aug;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0. Epub 2018 Aug 6.

25.

Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells.

Cromer MK, Vaidyanathan S, Ryan DE, Curry B, Lucas AB, Camarena J, Kaushik M, Hay SR, Martin RM, Steinfeld I, Bak RO, Dever DP, Hendel A, Bruhn L, Porteus MH.

Mol Ther. 2018 Oct 3;26(10):2431-2442. doi: 10.1016/j.ymthe.2018.06.002. Epub 2018 Jul 11.

26.

Gene Editing on Center Stage.

Bak RO, Gomez-Ospina N, Porteus MH.

Trends Genet. 2018 Aug;34(8):600-611. doi: 10.1016/j.tig.2018.05.004. Epub 2018 Jun 13. Review.

PMID:
29908711
27.

Role of human forkhead box P3 in early thymic maturation and peripheral T-cell homeostasis.

Santoni de Sio FR, Passerini L, Restelli S, Valente MM, Pramov A, Maccari ME, Sanvito F, Roncarolo MG, Porteus M, Bacchetta R.

J Allergy Clin Immunol. 2018 Dec;142(6):1909-1921.e9. doi: 10.1016/j.jaci.2018.03.015. Epub 2018 Apr 27. Erratum in: J Allergy Clin Immunol. 2019 Mar;143(3):1269.

PMID:
29705245
28.

MLL leukemia induction by t(9;11) chromosomal translocation in human hematopoietic stem cells using genome editing.

Schneidawind C, Jeong J, Schneidawind D, Kim IS, Duque-Afonso J, Wong SHK, Iwasaki M, Breese EH, Zehnder JL, Porteus M, Cleary ML.

Blood Adv. 2018 Apr 24;2(8):832-845. doi: 10.1182/bloodadvances.2017013748.

29.

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F, Miccio A.

Blood. 2018 Apr 26;131(17):1960-1973. doi: 10.1182/blood-2017-10-811505. Epub 2018 Mar 8.

PMID:
29519807
30.

CRISPR/Cas9 genome editing in human hematopoietic stem cells.

Bak RO, Dever DP, Porteus MH.

Nat Protoc. 2018 Feb;13(2):358-376. doi: 10.1038/nprot.2017.143. Epub 2018 Jan 25.

31.

Closing In on Treatment for Hemophilia B.

Porteus M.

N Engl J Med. 2017 Dec 7;377(23):2274-2275. doi: 10.1056/NEJMe1713735. No abstract available.

PMID:
29211662
32.

Genome Editing for the β-Hemoglobinopathies.

Porteus MH.

Adv Exp Med Biol. 2017;1013:203-217. doi: 10.1007/978-1-4939-7299-9_8.

PMID:
29127682
33.

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6.

Bak RO, Dever DP, Reinisch A, Cruz Hernandez D, Majeti R, Porteus MH.

Elife. 2017 Sep 28;6. pii: e27873. doi: 10.7554/eLife.27873. Erratum in: Elife. 2018 Nov 16;7:.

34.

The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation.

Dever DP, Porteus MH.

Curr Opin Hematol. 2017 Nov;24(6):481-488. doi: 10.1097/MOH.0000000000000385. Review.

35.

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors.

Bak RO, Porteus MH.

Cell Rep. 2017 Jul 18;20(3):750-756. doi: 10.1016/j.celrep.2017.06.064.

36.

Toward Responsible Human Genome Editing.

Hynes RO, Coller BS, Porteus M.

JAMA. 2017 May 9;317(18):1829-1830. doi: 10.1001/jama.2017.4548. No abstract available.

PMID:
28395003
37.

Genome Editing in Cardiovascular Biology.

Seeger T, Porteus M, Wu JC.

Circ Res. 2017 Mar 3;120(5):778-780. doi: 10.1161/CIRCRESAHA.116.310197. No abstract available.

38.

A Comprehensive TALEN-Based Knockout Library for Generating Human-Induced Pluripotent Stem Cell-Based Models for Cardiovascular Diseases.

Karakikes I, Termglinchan V, Cepeda DA, Lee J, Diecke S, Hendel A, Itzhaki I, Ameen M, Shrestha R, Wu H, Ma N, Shao NY, Seeger T, Woo N, Wilson KD, Matsa E, Porteus MH, Sebastiano V, Wu JC.

Circ Res. 2017 May 12;120(10):1561-1571. doi: 10.1161/CIRCRESAHA.116.309948. Epub 2017 Feb 28. Review.

39.

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH.

Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134. Epub 2016 Nov 7.

40.

Invasive Fungal Disease in Pediatric Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant.

Aftandilian C, Weinberg K, Willert J, Kharbanda S, Porteus M, Maldonado Y, Agarwal R.

J Pediatr Hematol Oncol. 2016 Oct;38(7):574-580.

PMID:
27658021
41.

Phosphorylation of residues inside the SNARE complex suppresses secretory vesicle fusion.

Malmersjö S, Di Palma S, Diao J, Lai Y, Pfuetzner RA, Wang AL, McMahon MA, Hayer A, Porteus M, Bodenmiller B, Brunger AT, Meyer T.

EMBO J. 2016 Aug 15;35(16):1810-21. doi: 10.15252/embj.201694071. Epub 2016 Jul 11.

42.

Knock-in editing: it functionally corrects!

Porteus MH.

Blood. 2016 May 26;127(21):2507-9. doi: 10.1182/blood-2016-03-703181. No abstract available.

43.

Ethical and regulatory aspects of genome editing.

Kohn DB, Porteus MH, Scharenberg AM.

Blood. 2016 May 26;127(21):2553-60. doi: 10.1182/blood-2016-01-678136. Epub 2016 Apr 6. Review.

PMID:
27053531
44.

Activation of proto-oncogenes by disruption of chromosome neighborhoods.

Hnisz D, Weintraub AS, Day DS, Valton AL, Bak RO, Li CH, Goldmann J, Lajoie BR, Fan ZP, Sigova AA, Reddy J, Borges-Rivera D, Lee TI, Jaenisch R, Porteus MH, Dekker J, Young RA.

Science. 2016 Mar 25;351(6280):1454-1458. doi: 10.1126/science.aad9024. Epub 2016 Mar 3.

45.

Mutations in the nuclear bile acid receptor FXR cause progressive familial intrahepatic cholestasis.

Gomez-Ospina N, Potter CJ, Xiao R, Manickam K, Kim MS, Kim KH, Shneider BL, Picarsic JL, Jacobson TA, Zhang J, He W, Liu P, Knisely AS, Finegold MJ, Muzny DM, Boerwinkle E, Lupski JR, Plon SE, Gibbs RA, Eng CM, Yang Y, Washington GC, Porteus MH, Berquist WE, Kambham N, Singh RJ, Xia F, Enns GM, Moore DD.

Nat Commun. 2016 Feb 18;7:10713. doi: 10.1038/ncomms10713.

46.

TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs.

Suzuki S, Sargent RG, Illek B, Fischer H, Esmaeili-Shandiz A, Yezzi MJ, Lee A, Yang Y, Kim S, Renz P, Qi Z, Yu J, Muench MO, Beyer AI, Guimarães AO, Ye L, Chang J, Fine EJ, Cradick TJ, Bao G, Rahdar M, Porteus MH, Shuto T, Kai H, Kan YW, Gruenert DC.

Mol Ther Nucleic Acids. 2016 Jan 5;5:e273. doi: 10.1038/mtna.2015.43.

47.

Towards a new era in medicine: therapeutic genome editing.

Porteus MH.

Genome Biol. 2015 Dec 22;16:286. doi: 10.1186/s13059-015-0859-y. Review.

48.

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.

Cell Rep. 2015 Dec 22;13(11):2412-2424. doi: 10.1016/j.celrep.2015.11.030. Epub 2015 Dec 10.

49.

Genome Editing: A New Approach to Human Therapeutics.

Porteus M.

Annu Rev Pharmacol Toxicol. 2016;56:163-90. doi: 10.1146/annurev-pharmtox-010814-124454. Epub 2015 Nov 9. Review.

PMID:
26566154
50.

Use of Genome Engineering to Create Patient Specific MLL Translocations in Primary Human Hematopoietic Stem and Progenitor Cells.

Breese EH, Buechele C, Dawson C, Cleary ML, Porteus MH.

PLoS One. 2015 Sep 9;10(9):e0136644. doi: 10.1371/journal.pone.0136644. eCollection 2015.

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