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Items: 1 to 50 of 90

1.

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.

2.

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
3.

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.

4.

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.

5.

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.

6.

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
7.

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.

8.

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.

9.

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
10.

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
11.

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
12.

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.

13.

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.

PMID:
30424953
14.

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
15.

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.

16.

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.

17.

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.

18.

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.

19.

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.

20.

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
21.

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.

22.

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
23.

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:.

24.

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.

25.

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.

26.

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.

27.

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.

28.

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.

29.

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
30.

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.

31.

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.

32.

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.

33.

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.

34.

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.

35.

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.

Hendel A, Bak RO, Clark JT, Kennedy AB, Ryan DE, Roy S, Steinfeld I, Lunstad BD, Kaiser RJ, Wilkens AB, Bacchetta R, Tsalenko A, Dellinger D, Bruhn L, Porteus MH.

Nat Biotechnol. 2015 Sep;33(9):985-989. doi: 10.1038/nbt.3290. Epub 2015 Jun 29.

36.

Genome Editing of the Blood: Opportunities and Challenges.

Porteus MH.

Curr Stem Cell Rep. 2015 Mar 1;1(1):23-30.

37.

Genome editing of the germline: broadening the discussion.

Porteus MH, Dann CT.

Mol Ther. 2015 Jun;23(6):980-982. doi: 10.1038/mt.2015.83.

38.

Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin.

Wienert B, Funnell AP, Norton LJ, Pearson RC, Wilkinson-White LE, Lester K, Vadolas J, Porteus MH, Matthews JM, Quinlan KG, Crossley M.

Nat Commun. 2015 May 14;6:7085. doi: 10.1038/ncomms8085.

PMID:
25971621
39.

Genome editing technologies: defining a path to clinic.

Corrigan-Curay J, O'Reilly M, Kohn DB, Cannon PM, Bao G, Bushman FD, Carroll D, Cathomen T, Joung JK, Roth D, Sadelain M, Scharenberg AM, von Kalle C, Zhang F, Jambou R, Rosenthal E, Hassani M, Singh A, Porteus MH.

Mol Ther. 2015 May;23(5):796-806. doi: 10.1038/mt.2015.54. No abstract available.

40.

Quantifying on- and off-target genome editing.

Hendel A, Fine EJ, Bao G, Porteus MH.

Trends Biotechnol. 2015 Feb;33(2):132-40. doi: 10.1016/j.tibtech.2014.12.001. Epub 2015 Jan 13. Review.

41.

Genome editing in mouse spermatogonial stem/progenitor cells using engineered nucleases.

Fanslow DA, Wirt SE, Barker JC, Connelly JP, Porteus MH, Dann CT.

PLoS One. 2014 Nov 19;9(11):e112652. doi: 10.1371/journal.pone.0112652. eCollection 2014.

42.

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA.

Nature. 2015 Jan 15;517(7534):360-4. doi: 10.1038/nature13864. Epub 2014 Oct 29.

43.

Gene/cell therapy approaches for Immune Dysregulation Polyendocrinopathy Enteropathy X-linked syndrome.

Passerini L, Santoni de Sio FR, Porteus MH, Bacchetta R.

Curr Gene Ther. 2014;14(6):422-8. Review.

44.

Genome editing of mouse fibroblasts by homologous recombination for sustained secretion of PDGF-B and augmentation of wound healing.

Barker JC, Barker AD, Bills J, Huang J, Wight-Carter M, Delgado I, Noble DL, Huang LJ, Porteus MH, Davis KE.

Plast Reconstr Surg. 2014 Sep;134(3):389e-401e. doi: 10.1097/PRS.0000000000000427.

PMID:
25158716
45.

Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ.

N Engl J Med. 2014 Jul 31;371(5):434-46. doi: 10.1056/NEJMoa1401177.

46.

Lentiviral and targeted cellular barcoding reveals ongoing clonal dynamics of cell lines in vitro and in vivo.

Porter SN, Baker LC, Mittelman D, Porteus MH.

Genome Biol. 2014 May 30;15(5):R75. doi: 10.1186/gb-2014-15-5-r75.

47.

Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing.

Hendel A, Kildebeck EJ, Fine EJ, Clark J, Punjya N, Sebastiano V, Bao G, Porteus MH.

Cell Rep. 2014 Apr 10;7(1):293-305. doi: 10.1016/j.celrep.2014.02.040. Epub 2014 Mar 27.

48.

SAPTA: a new design tool for improving TALE nuclease activity.

Lin Y, Fine EJ, Zheng Z, Antico CJ, Voit RA, Porteus MH, Cradick TJ, Bao G.

Nucleic Acids Res. 2014 Apr;42(6):e47. doi: 10.1093/nar/gkt1363. Epub 2014 Jan 16.

49.

Nuclease-mediated gene editing by homologous recombination of the human globin locus.

Voit RA, Hendel A, Pruett-Miller SM, Porteus MH.

Nucleic Acids Res. 2014 Jan;42(2):1365-78. doi: 10.1093/nar/gkt947. Epub 2013 Oct 23.

50.

An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level.

Bauer DE, Kamran SC, Lessard S, Xu J, Fujiwara Y, Lin C, Shao Z, Canver MC, Smith EC, Pinello L, Sabo PJ, Vierstra J, Voit RA, Yuan GC, Porteus MH, Stamatoyannopoulos JA, Lettre G, Orkin SH.

Science. 2013 Oct 11;342(6155):253-7. doi: 10.1126/science.1242088.

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