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Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.

Humbert O, Radtke S, Samuelson C, Carrillo RR, Perez AM, Reddy SS, Lux C, Pattabhi S, Schefter LE, Negre O, Lee CM, Bao G, Adair JE, Peterson CW, Rawlings DJ, Scharenberg AM, Kiem HP.

Sci Transl Med. 2019 Jul 31;11(503). pii: eaaw3768. doi: 10.1126/scitranslmed.aaw3768.


In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods.

Pattabhi S, Lotti SN, Berger MP, Singh S, Lux CT, Jacoby K, Lee C, Negre O, Scharenberg AM, Rawlings DJ.

Mol Ther Nucleic Acids. 2019 Sep 6;17:277-288. doi: 10.1016/j.omtn.2019.05.025. Epub 2019 Jun 7.


Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy.

Humbert O, Chan F, Rajawat YS, Torgerson TR, Burtner CR, Hubbard NW, Humphrys D, Norgaard ZK, O'Donnell P, Adair JE, Trobridge GD, Scharenberg AM, Felsburg PJ, Rawlings DJ, Kiem HP.

Blood Adv. 2018 May 8;2(9):987-999. doi: 10.1182/bloodadvances.2018016451.


Correction: The BRCT Domain of PARP-1 Is Required for Immunoglobulin Gene Conversion.

Paddock MN, Buelow BD, Takeda S, Scharenberg AM.

PLoS Biol. 2018 Mar 1;16(3):e1002621. doi: 10.1371/journal.pbio.1002621. eCollection 2018 Mar.


Therapeutic Gene Editing Safety and Specificity.

Lux CT, Scharenberg AM.

Hematol Oncol Clin North Am. 2017 Oct;31(5):787-795. doi: 10.1016/j.hoc.2017.05.002. Epub 2017 Jun 29. Review.


Homology-Directed Recombination for Enhanced Engineering of Chimeric Antigen Receptor T Cells.

Hale M, Lee B, Honaker Y, Leung WH, Grier AE, Jacobs HM, Sommer K, Sahni J, Jackson SW, Scharenberg AM, Astrakhan A, Rawlings DJ.

Mol Ther Methods Clin Dev. 2017 Jan 10;4:192-203. doi: 10.1016/j.omtm.2016.12.008. eCollection 2017 Mar 17.


Characterization of homing endonuclease binding and cleavage specificities using yeast surface display SELEX (YSD-SELEX).

Jacoby K, Lambert AR, Scharenberg AM.

Nucleic Acids Res. 2017 Feb 17;45(3):e11. doi: 10.1093/nar/gkw864.


Engineering HIV-Resistant, Anti-HIV Chimeric Antigen Receptor T Cells.

Hale M, Mesojednik T, Romano Ibarra GS, Sahni J, Bernard A, Sommer K, Scharenberg AM, Rawlings DJ, Wagner TA.

Mol Ther. 2017 Mar 1;25(3):570-579. doi: 10.1016/j.ymthe.2016.12.023. Epub 2017 Jan 28.


High Efficiency CRISPR/Cas9-mediated Gene Editing in Primary Human T-cells Using Mutant Adenoviral E4orf6/E1b55k "Helper" Proteins.

Gwiazda KS, Grier AE, Sahni J, Burleigh SM, Martin U, Yang JG, Popp NA, Krutein MC, Khan IF, Jacoby K, Jensen MC, Rawlings DJ, Scharenberg AM.

Mol Ther. 2016 Sep 29;24(9):1570-80. doi: 10.1038/mt.2016.105. Epub 2016 May 16.


Indirect DNA Sequence Recognition and Its Impact on Nuclease Cleavage Activity.

Lambert AR, Hallinan JP, Shen BW, Chik JK, Bolduc JM, Kulshina N, Robins LI, Kaiser BK, Jarjour J, Havens K, Scharenberg AM, Stoddard BL.

Structure. 2016 Jun 7;24(6):862-73. doi: 10.1016/j.str.2016.03.024. Epub 2016 Apr 28.


pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences.

Grier AE, Burleigh S, Sahni J, Clough CA, Cardot V, Choe DC, Krutein MC, Rawlings DJ, Jensen MC, Scharenberg AM, Jacoby K.

Mol Ther Nucleic Acids. 2016 Apr 19;5:e306. doi: 10.1038/mtna.2016.21.


X-Linked Severe Combined Immunodeficiency.

Allenspach E, Rawlings DJ, Scharenberg AM.

In: Adam MP, Ardinger HH, Pagon RA, Wallace SE, Bean LJH, Stephens K, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2019.
2003 Aug 26 [updated 2016 Apr 14].


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.


Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome.

Hubbard N, Hagin D, Sommer K, Song Y, Khan I, Clough C, Ochs HD, Rawlings DJ, Scharenberg AM, Torgerson TR.

Blood. 2016 May 26;127(21):2513-22. doi: 10.1182/blood-2015-11-683235. Epub 2016 Feb 22.


Digital detection of endonuclease mediated gene disruption in the HIV provirus.

Sedlak RH, Liang S, Niyonzima N, De Silva Feelixge HS, Roychoudhury P, Greninger AL, Weber ND, Boissel S, Scharenberg AM, Cheng A, Magaret A, Bumgarner R, Stone D, Jerome KR.

Sci Rep. 2016 Feb 2;6:20064. doi: 10.1038/srep20064.


Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases.

Osborn MJ, Webber BR, Knipping F, Lonetree CL, Tennis N, DeFeo AP, McElroy AN, Starker CG, Lee C, Merkel S, Lund TC, Kelly-Spratt KS, Jensen MC, Voytas DF, von Kalle C, Schmidt M, Gabriel R, Hippen KL, Miller JS, Scharenberg AM, Tolar J, Blazar BR.

Mol Ther. 2016 Mar;24(3):570-81. doi: 10.1038/mt.2015.197. Epub 2015 Oct 27.


Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template.

Sather BD, Romano Ibarra GS, Sommer K, Curinga G, Hale M, Khan IF, Singh S, Song Y, Gwiazda K, Sahni J, Jarjour J, Astrakhan A, Wagner TA, Scharenberg AM, Rawlings DJ.

Sci Transl Med. 2015 Sep 30;7(307):307ra156. doi: 10.1126/scitranslmed.aac5530.


Multiplex Genome-Edited T-cell Manufacturing Platform for "Off-the-Shelf" Adoptive T-cell Immunotherapies.

Poirot L, Philip B, Schiffer-Mannioui C, Le Clerre D, Chion-Sotinel I, Derniame S, Potrel P, Bas C, Lemaire L, Galetto R, Lebuhotel C, Eyquem J, Cheung GW, Duclert A, Gouble A, Arnould S, Peggs K, Pule M, Scharenberg AM, Smith J.

Cancer Res. 2015 Sep 15;75(18):3853-64. doi: 10.1158/0008-5472.CAN-14-3321. Epub 2015 Jul 16.


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.


Assembly and characterization of megaTALs for hyperspecific genome engineering applications.

Boissel S, Scharenberg AM.

Methods Mol Biol. 2015;1239:171-96. doi: 10.1007/978-1-4939-1862-1_9.


Progressive engineering of a homing endonuclease genome editing reagent for the murine X-linked immunodeficiency locus.

Wang Y, Khan IF, Boissel S, Jarjour J, Pangallo J, Thyme S, Baker D, Scharenberg AM, Rawlings DJ.

Nucleic Acids Res. 2014 Jun;42(10):6463-75. doi: 10.1093/nar/gku224. Epub 2014 Mar 25.


Intravenous injection of a foamy virus vector to correct canine SCID-X1.

Burtner CR, Beard BC, Kennedy DR, Wohlfahrt ME, Adair JE, Trobridge GD, Scharenberg AM, Torgerson TR, Rawlings DJ, Felsburg PJ, Kiem HP.

Blood. 2014 Jun 5;123(23):3578-84. doi: 10.1182/blood-2013-11-538926. Epub 2014 Mar 18.


Engineering and flow-cytometric analysis of chimeric LAGLIDADG homing endonucleases from homologous I-OnuI-family enzymes.

Baxter SK, Scharenberg AM, Lambert AR.

Methods Mol Biol. 2014;1123:191-221. doi: 10.1007/978-1-62703-968-0_14.


Homing endonuclease target determination using SELEX adapted for yeast surface display.

Jacoby K, Scharenberg AM.

Methods Mol Biol. 2014;1123:165-90. doi: 10.1007/978-1-62703-968-0_13.


In vitro Inactivation of Latent HSV by Targeted Mutagenesis Using an HSV-specific Homing Endonuclease.

Aubert M, Boyle NM, Stone D, Stensland L, Huang ML, Magaret AS, Galetto R, Rawlings DJ, Scharenberg AM, Jerome KR.

Mol Ther Nucleic Acids. 2014 Feb 4;3:e146. doi: 10.1038/mtna.2013.75.


megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering.

Boissel S, Jarjour J, Astrakhan A, Adey A, Gouble A, Duchateau P, Shendure J, Stoddard BL, Certo MT, Baker D, Scharenberg AM.

Nucleic Acids Res. 2014 Feb;42(4):2591-601. doi: 10.1093/nar/gkt1224. Epub 2013 Nov 26.


Novel fluorescent genome editing reporters for monitoring DNA repair pathway utilization at endonuclease-induced breaks.

Kuhar R, Gwiazda KS, Humbert O, Mandt T, Pangallo J, Brault M, Khan I, Maizels N, Rawlings DJ, Scharenberg AM, Certo MT.

Nucleic Acids Res. 2014 Jan;42(1):e4. doi: 10.1093/nar/gkt872. Epub 2013 Oct 10.


Pillars article: Recruitment of tyrosine phosphatase HCP by the killer cell inhibitory receptor. Immunity. 1996. 4: 77-85.

Burshtyn DN, Scharenberg AM, Wagtmann N, Rajagopalan S, Berrada K, Yi T, Kinet JP, Long EO.

J Immunol. 2013 Oct 1;191(7):3491-9. No abstract available.


Transient receptor potential melastatin 7 is involved in oestrogen receptor-negative metastatic breast cancer cells migration through its kinase domain.

Guilbert A, Gautier M, Dhennin-Duthille I, Rybarczyk P, Sahni J, Sevestre H, Scharenberg AM, Ouadid-Ahidouch H.

Eur J Cancer. 2013 Nov;49(17):3694-707. doi: 10.1016/j.ejca.2013.07.008. Epub 2013 Aug 1.


Genome engineering with TAL-effector nucleases and alternative modular nuclease technologies.

Scharenberg AM, Duchateau P, Smith J.

Curr Gene Ther. 2013 Aug;13(4):291-303. Review.


The SLC41 family of MgtE-like magnesium transporters.

Sahni J, Scharenberg AM.

Mol Aspects Med. 2013 Apr-Jun;34(2-3):620-8. doi: 10.1016/j.mam.2012.05.012. Review.


Characterization of three loci for homologous gene targeting and transgene expression.

Eyquem J, Poirot L, Galetto R, Scharenberg AM, Smith J.

Biotechnol Bioeng. 2013 Aug;110(8):2225-35. doi: 10.1002/bit.24892. Epub 2013 May 2.


Flow cytometric assays for interrogating LAGLIDADG homing endonuclease DNA-binding and cleavage properties.

Baxter SK, Lambert AR, Scharenberg AM, Jarjour J.

Methods Mol Biol. 2013;978:45-61. doi: 10.1007/978-1-62703-293-3_4.


The B. subtilis MgtE magnesium transporter can functionally compensate TRPM7-deficiency in vertebrate B-cells.

Sahni J, Song Y, Scharenberg AM.

PLoS One. 2012;7(9):e44452. doi: 10.1371/journal.pone.0044452. Epub 2012 Sep 6.


Coupling endonucleases with DNA end-processing enzymes to drive gene disruption.

Certo MT, Gwiazda KS, Kuhar R, Sather B, Curinga G, Mandt T, Brault M, Lambert AR, Baxter SK, Jacoby K, Ryu BY, Kiem HP, Gouble A, Paques F, Rawlings DJ, Scharenberg AM.

Nat Methods. 2012 Oct;9(10):973-5. doi: 10.1038/nmeth.2177. Epub 2012 Sep 2.


Engineering domain fusion chimeras from I-OnuI family LAGLIDADG homing endonucleases.

Baxter S, Lambert AR, Kuhar R, Jarjour J, Kulshina N, Parmeggiani F, Danaher P, Gano J, Baker D, Stoddard BL, Scharenberg AM.

Nucleic Acids Res. 2012 Sep;40(16):7985-8000. doi: 10.1093/nar/gks502. Epub 2012 Jun 7.


Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects.

Ramirez CL, Certo MT, Mussolino C, Goodwin MJ, Cradick TJ, McCaffrey AP, Cathomen T, Scharenberg AM, Joung JK.

Nucleic Acids Res. 2012 Jul;40(12):5560-8. doi: 10.1093/nar/gks179. Epub 2012 Feb 28.


Expanding LAGLIDADG endonuclease scaffold diversity by rapidly surveying evolutionary sequence space.

Jacoby K, Metzger M, Shen BW, Certo MT, Jarjour J, Stoddard BL, Scharenberg AM.

Nucleic Acids Res. 2012 Jun;40(11):4954-64. doi: 10.1093/nar/gkr1303. Epub 2012 Feb 14.


Novel reporter systems for facile evaluation of I-SceI-mediated genome editing.

Muñoz NM, Beard BC, Ryu BY, Luche RM, Trobridge GD, Rawlings DJ, Scharenberg AM, Kiem HP.

Nucleic Acids Res. 2012 Jan;40(2):e14. doi: 10.1093/nar/gkr897. Epub 2011 Nov 21.


Tapping natural reservoirs of homing endonucleases for targeted gene modification.

Takeuchi R, Lambert AR, Mak AN, Jacoby K, Dickson RJ, Gloor GB, Scharenberg AM, Edgell DR, Stoddard BL.

Proc Natl Acad Sci U S A. 2011 Aug 9;108(32):13077-82. doi: 10.1073/pnas.1107719108. Epub 2011 Jul 22.


Tracking genome engineering outcome at individual DNA breakpoints.

Certo MT, Ryu BY, Annis JE, Garibov M, Jarjour J, Rawlings DJ, Scharenberg AM.

Nat Methods. 2011 Jul 10;8(8):671-6. doi: 10.1038/nmeth.1648.


SLC41A1 Mg(2+) transport is regulated via Mg(2+)-dependent endosomal recycling through its N-terminal cytoplasmic domain.

Mandt T, Song Y, Scharenberg AM, Sahni J.

Biochem J. 2011 Oct 1;439(1):129-39. doi: 10.1042/BJ20110807.


Successful targeting and disruption of an integrated reporter lentivirus using the engineered homing endonuclease Y2 I-AniI.

Aubert M, Ryu BY, Banks L, Rawlings DJ, Scharenberg AM, Jerome KR.

PLoS One. 2011 Feb 9;6(2):e16825. doi: 10.1371/journal.pone.0016825.


Competition between PARP-1 and Ku70 control the decision between high-fidelity and mutagenic DNA repair.

Paddock MN, Bauman AT, Higdon R, Kolker E, Takeda S, Scharenberg AM.

DNA Repair (Amst). 2011 Mar 7;10(3):338-43. doi: 10.1016/j.dnarep.2010.12.005. Epub 2011 Jan 20.


Efficacy and safety of a new immunoglobulin G product, Gammaplex(®), in primary immunodeficiency diseases.

Moy JN, Scharenberg AM, Stein MR, Suez D, Roberts RL, Levy RJ, Ballow M, Fasano MB, Dash CH, Leach SJ.

Clin Exp Immunol. 2010 Dec;162(3):510-5. doi: 10.1111/j.1365-2249.2010.04247.x.


TRPM7 regulates quiescent/proliferative metabolic transitions in lymphocytes.

Sahni J, Tamura R, Sweet IR, Scharenberg AM.

Cell Cycle. 2010 Sep 1;9(17):3565-74. doi: 10.4161/cc.9.17.12798. Epub 2010 Sep 25.


The BRCT domain of PARP-1 is required for immunoglobulin gene conversion.

Paddock MN, Buelow BD, Takeda S, Scharenberg AM.

PLoS Biol. 2010 Jul 20;8(7):e1000428. doi: 10.1371/journal.pbio.1000428. Erratum in: PLoS Biol. 2018 Mar 1;16(3):e1002621.


Stable hematopoietic cell engraftment after low-intensity nonmyeloablative conditioning in patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome.

Burroughs LM, Torgerson TR, Storb R, Carpenter PA, Rawlings DJ, Sanders J, Scharenberg AM, Skoda-Smith S, Englund J, Ochs HD, Woolfrey AE.

J Allergy Clin Immunol. 2010 Nov;126(5):1000-5. doi: 10.1016/j.jaci.2010.05.021.


Exploitation of binding energy for catalysis and design.

Thyme SB, Jarjour J, Takeuchi R, Havranek JJ, Ashworth J, Scharenberg AM, Stoddard BL, Baker D.

Nature. 2009 Oct 29;461(7268):1300-4. doi: 10.1038/nature08508.


Zinc-finger nucleases: a powerful tool for genetic engineering of animals.

Rémy S, Tesson L, Ménoret S, Usal C, Scharenberg AM, Anegon I.

Transgenic Res. 2010 Jun;19(3):363-71. doi: 10.1007/s11248-009-9323-7. Epub 2009 Sep 26. Review.


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