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Items: 1 to 50 of 76

1.

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.

2.

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.

3.

SCID genotype and 6-month post-transplant CD4 count predict survival and immune recovery: a PIDTC retrospective study.

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 A, 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 Aug 28. pii: blood-2018-03-840702. doi: 10.1182/blood-2018-03-840702. [Epub ahead of print]

PMID:
30154114
4.

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.

PMID:
30082871
5.

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 Jul 10. pii: S1525-0016(18)30261-2. doi: 10.1016/j.ymthe.2018.06.002. [Epub ahead of print]

PMID:
30005866
6.

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
7.

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.

8.

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
9.

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.

10.

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.

PMID:
28806273
11.

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.

12.

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.

13.

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.

14.

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.

15.

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.

16.

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.

17.

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.

18.

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.

19.

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.

20.

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.

21.

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.

22.

Genome Editing of the Blood: Opportunities and Challenges.

Porteus MH.

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

23.

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.

24.

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
25.

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.

26.

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.

27.

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.

28.

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.

29.

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.

30.

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
31.

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.

32.

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.

33.

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.

34.

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.

35.

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.

36.

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.

37.

Receptor-mediated delivery of engineered nucleases for genome modification.

Chen Z, Jaafar L, Agyekum DG, Xiao H, Wade MF, Kumaran RI, Spector DL, Bao G, Porteus MH, Dynan WS, Meiler SE.

Nucleic Acids Res. 2013 Oct;41(19):e182. doi: 10.1093/nar/gkt710. Epub 2013 Aug 16.

38.

Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification.

Wilson KA, McEwen AE, Pruett-Miller SM, Zhang J, Kildebeck EJ, Porteus MH.

Mol Ther Nucleic Acids. 2013 Apr 30;2:e88. doi: 10.1038/mtna.2013.13.

39.

Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus.

Wilson KA, Chateau ML, Porteus MH.

Mol Ther Nucleic Acids. 2013 Apr 23;2:e87. doi: 10.1038/mtna.2013.12.

40.

A crisper look at genome editing: RNA-guided genome modification.

Damian M, Porteus MH.

Mol Ther. 2013 Apr;21(4):720-2. doi: 10.1038/mt.2013.46. No abstract available.

41.
42.

Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors.

Voit RA, McMahon MA, Sawyer SL, Porteus MH.

Mol Ther. 2013 Apr;21(4):786-95. doi: 10.1038/mt.2012.284. Epub 2013 Jan 29.

43.

Engineering the immune system to cure genetic diseases, HIV, and cancer.

Porteus MH, Fischer A.

Curr Opin Immunol. 2012 Oct;24(5):576-9. doi: 10.1016/j.coi.2012.09.004. No abstract available.

PMID:
23084086
44.

Development of nuclease-mediated site-specific genome modification.

Wirt SE, Porteus MH.

Curr Opin Immunol. 2012 Oct;24(5):609-16. doi: 10.1016/j.coi.2012.08.005. Epub 2012 Sep 13. Review.

PMID:
22981684
45.

Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs.

Ellis BL, Hirsch ML, Porter SN, Samulski RJ, Porteus MH.

Gene Ther. 2013 Jan;20(1):35-42. doi: 10.1038/gt.2011.211. Epub 2012 Jan 19.

46.

Viral single-strand DNA induces p53-dependent apoptosis in human embryonic stem cells.

Hirsch ML, Fagan BM, Dumitru R, Bower JJ, Yadav S, Porteus MH, Pevny LH, Samulski RJ.

PLoS One. 2011;6(11):e27520. doi: 10.1371/journal.pone.0027520. Epub 2011 Nov 17.

47.

Creating higher titer lentivirus with caffeine.

Ellis BL, Potts PR, Porteus MH.

Hum Gene Ther. 2011 Jan;22(1):93-100. doi: 10.1089/hum.2010.068. Epub 2010 Dec 12.

48.

MOF and histone H4 acetylation at lysine 16 are critical for DNA damage response and double-strand break repair.

Sharma GG, So S, Gupta A, Kumar R, Cayrou C, Avvakumov N, Bhadra U, Pandita RK, Porteus MH, Chen DJ, Cote J, Pandita TK.

Mol Cell Biol. 2010 Jul;30(14):3582-95. doi: 10.1128/MCB.01476-09. Epub 2010 May 17.

49.

Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair.

Hirsch ML, Green L, Porteus MH, Samulski RJ.

Gene Ther. 2010 Sep;17(9):1175-80. doi: 10.1038/gt.2010.65. Epub 2010 May 13.

50.

Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic disease.

Connelly JP, Barker JC, Pruett-Miller S, Porteus MH.

Mol Ther. 2010 Jun;18(6):1103-10. doi: 10.1038/mt.2010.57. Epub 2010 Apr 13.

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