Selected Publications
Peer-reviewed Publications
Porteus MH, Bulfone A, Ciaranello RD, et al. Isolation and characterization of a novel cDNA clone encoding a homeodomain that is developmentally regulated in the ventral forebrain. Neuron 1991 Aug;7(2):221-9. Erratum in: Neuron 1992 Jul;9(1):187.
Porteus MH, Brice AE, Bulfone A, et al. Isolation and characterization of a library of cDNA clones that are preferentially expressed in the embryonic telencephalon. Brain Res Mol Brain Res 1992 Jan;12(1-3):7-22.
Porteus MH, Bulfone A, Liu JK, et al. DLX-2, MASH-1, and MAP-2 expression and bromodeoxyuridine incorporation define molecularly distinct cell populations in the embryonic mouse forebrain. J Neurosci 1994 Nov;14(11 Pt 1):6370-83.
Porteus MH, Narkool P, Neuberg D, et al. Characteristics and outcome of children with Beckwith-Wiedemann syndrome and Wilms' tumor: a report from the National Wilms Tumor Study Group. J Clin Oncol 2000 May;18(10):2026-31.
Porteus MH*, Cathomen T, Weitzman MD, et al. Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks. Mol Cell Biol 2003 May;23(10):3558-65.
Porteus MH*, Baltimore D. Chimeric nucleases stimulate gene targeting in human cells. Science 2003 May 2;300(5620):763.
Urnov, FD, Miller, JC, Lee, Y-L, Beausejour, CM, Rock, J, Augustus, S, Jamieson, AC, Porteus, MH*, Gregory, P.D., and Holmes, M.C. (2005). Highly Efficient Endogenous Human Gene Correction Using Designed Zinc Finger Nucleases. Nature. 435, 646-51.
Porteus, MH*. Mammalian Gene Targeting with Designed Zinc Finger Nucleases. Mol Therapy 2006 Feb; 13(2)): 438-446.
Potts, PR, Porteus, MH*, and Yu, H.* Human SMC5/6 Complex Promotes Sister-Chromatid Homologous Recombination by Recruiting the SMC1/3 Cohesin Complex to Double-Strand Breaks. EMBO 2006, 25: 3377-3388.
Wright DA, Thibodeau-Beganny S, Sander JD, Winfrey RJ, Hirsh AS, Eichtinger M, Fu F, Porteus MH, Dobbs D, Voytas DF, Joung JK. Standardized reagents and protocols for engineering zinc finger nucleases by modular assembly. Nat Protoc. 2006;1(3):1637-52.
Pruett-Miller, SM, Connelly, JP, Maeder, ML, Joung, JK, and Porteus, MH*. Comparison of Zinc Finger Nucleases for use in Gene Targeting in Mammalian Cells. Molecular Therapy 2008 16: 707-717.
Dann CT, Alvarado AL, Molyneux LA, Denard BS, Garbers DL, Porteus MH*. Spermatogonial stem cell self-renewal requires OCT4, a factor downregulated during retinoic acid-induced differentiation. Stem Cells. 2008 Nov;26(11):2928-37.
Maeder, ML, Thibodeau-Beganny, S., Osiak, A, Wright, DA, Anthony, RM, Eichtinger, M, Jiang, T, Foley, JE, Winfrey, RJ, Townsend, JA, Unger-Wallace, E, Sander, JD, Muller-Lerch, F, Fu, F, Pearlberg, J, Gobel, C, Dassie, JD, Pruett-Miller, SM, Porteus, MH, Sgroi, DC, Iafrate, AJ, Dobbs, D, McCray, PB, Cathomen, T, Voytas, DF, and Joung, KJ. Rapid “open-source” engineering of customized zinc-finger nucleases for highly efficient gene modification. Molecular Cell 2008 31: 294-301.
Pruett-Miller, S, Reading, DW, Porter, SN, and Porteus, MH* Attenuation of Zinc Finger Nuclease Toxicity by Small Molecule Regulation of Protein Expression. PLoS Genetics 2009 5(2): e1000376.
Zou, J, Maeder, ML, Mali, P, Pruett-Miller, SM, Thibodeau-Beganny, S, Chou, B-K, Chen, G, Ye, Z, Park, I-H, Daley, GQ, Porteus, MH*, Joung, JK*, and Cheng, L* Gene Targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells. Cell Stem Cells 2009 5: 97-110.
Connelly, JP, Barker, J, Pruett-Miller, S, and Porteus, MH.* Gene Correction by Homologous Recombination with Zinc Finger Nucleases in Primary Cells from a Mouse Model of a Generic Recessive Genetic Disease. Molecular Therapy 2010 18: 1103-1110.
Hirsch, M, Green, L, Porteus, MH, and Samulski, J. Self-complementary AAV Mediates Gene Targeting and Enhances Endonuclease Delivery for Double-Strand Break Repair. Gene Therapy 2010, 17: 1175-1180.
Sharma, GG, So, S, Gupta, A, Kumar, R, Cayrou, C, Avvakumov, N, Bhadra, U, Pandita, RK, Porteus, MH, Chen, DJ, Cote, J, Pandita, TK. MOF and histone H4 acetylation at lysine 16 are critical for DNA damage response and double-strand break repair. Mol Cell Biol. 2010 Jul;30(14):3582-95.
Ellis, BL, Potts, PR, Porteus, MH.* Creating Higher Titer Lentivirus using Caffeine. Hum Gene Ther 2010, 22: 93-100.
Hirsch, ML, Fagan, BM, Dumitru, R, Bower, JJ, Yadav, S, Porteus, MH, Pevny, LH, Samulski, RJ. Viral single-strand DNA induces p53-dependent apoptosis in human embryonic stem cells. PLoS One. 2011;6(11).
Ellis, BL, Hirsch, ML, Porter, SN, Samulski, RJ, Porteus, MH*. Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs. Gene Ther. 2012 Jan.
Ellis, BL, Barker, JC, Hirsch, ML, Connelly, JP, Steninger, RJ, and Porteus, MH*. A Survey of ex vivo Transduction Efficiency of Mammalian Primary Cells and Cell Lines with Nine Natural (AAV1-9) and One Engineered AAV Serotype. Manuscript Submitted to PLoS One.
Voit, RA, McMahon, MA, Sawyer, SL, and Porteus MH. Generation of an HIV resistant T-cell line by targered “stacking” of restriction factors. Mol Ther. 2013 21: 786-795.
Wilson, KA, Chateau, ML, and Porteus, MH*. Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus. Mol Ther Nucleic Acids. 2013 Apr 23; e87.
Wilson, KA, McEwen, AE, Preutt-Miller, SM, Zhang, J., Kildebeck, EJ, and Porteus, MH*. Expanding the repertoire of target sites for zinc finger nuclease mediated genome modification. Mol Ther Nucleic Acids. 2013 Apr 30;2:e88.
Kwan, A, Church, JA, Cowan, MJ, Agarwal, R, Kapoor, N., Kohn, DB, Lewis, DB, McGhee, SA, Moore, TB, Stiehm, ER, Porteus, M, Aznar, CP, Currier, R, Lorey, F. and Puck, JM. Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: Results of the first 2 years. J Allergt Clin Immunol. 2013 132: 140-150.
Voit, RA, Hendel, A, Pruett-Miller, SM, Porteus, MH*. 2012. Therapeutic gene editing and the generation of endogenous fluorescent reporters at the human globin loci. Manuscript Submitted.
Reviews and News
Porteus MH*, Carroll D. Gene targeting using zinc finger nucleases. Nat Biotechnol. 2005 Aug;23(8):967-73.
Durai S, Mani M, Kandavelou K, Wu J, Porteus MH, Chandrasegaran S. Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells. Nucleic Acids Res. 2005 Oct 26;33(18):5978-90.
Porteus MH*, Connelly JP, Pruett SM. A look to future directions in gene therapy research for monogenic diseases. PLoS Genet. 2006 Sep 29;2(9):e133.
Porteus M*. Using homologous recombination to manipulate the genome of human somatic cells. Biotechnol Genet Eng Rev. 2007;24:195-212.
Porteus MH*. Plant biotechnology: Zinc fingers on target. Nature. 2009 May 21;459(7245):337-8.
Ellis J, Baum C, Benvenisty N, Mostoslavsky G, Okano H, Stanford WL, Porteus M, Sadelain M. Benefits of utilizing gene-modified iPSCs for clinical applications. Cell Stem Cell. 2010 Oct 8;7(4):429-30.
Porteus M*. Creating zinc finger nucleases using a modular-assembly approach. Cold Spring Harb Protoc. 2010 Dec 1;2010(12).
Porteus M*. Testing a three-finger zinc finger nuclease using a GFP reporter system. Cold Spring Harb Protoc. 2010 Dec 1;2010(12).
Porteus M*. Translating the lessons from gene therapy to the development of regenerative medicine. Mol Ther. 2011 Mar;19(3):439-41.
Porteus M*. Seeing the light: integrating genome engineering with double-strand break repair. Nat Methods. 2011 Jul 28;8(8):628-30.
Cheng L, Blazar B, High K, Porteus M*. Zinc fingers hit off target. Nat Med. 2011 Oct 11;17(10):1192-3.
McMahon MA, Rahdar M, Porteus M*. Gene editing: not just for translation anymore. Nat Methods. 2011 Dec 28;9(1):28-31.
Porteus M*. Homologous recombination-based gene therapy for the primary immunodeficiencies. Ann N Y Acad Sci. 2011 Dec;1246:131-40.
Wirt SE, Porteus MH*. Development of nuclease-mediated site-specific genome modification. Curr Opin Immunol. 2012 Oct;24(5):609-16.
Kildebeck E, Checketts J, Porteus M*. Gene therapy for primary immunodeficiencies. Curr Opin Pediatr. 2012 Dec;24(6):731-8.
Porteus MH*, Fischer A. Engineering the immune system to cure genetic diseases, HIV, and cancer. Curr Opin Immunol. 2012 Oct;24(5):576-9.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther. 2013 Feb;21(2):266-8.
Damian M, Porteus MH*. A crisper look at genome editing: RNA-guided genome modification. Mol Ther. 2013 Apr;21(4):720-2.
(in chronological order, * denotes corresponding author)