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Items: 1 to 50 of 278

1.

Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes.

Ozog S, Timberlake ND, Hermann K, Garijo O, Haworth KG, Shi G, Glinkerman CM, Schefter LE, D'Souza S, Simpson E, Sghia-Hughes G, Carillo RR, Boger DL, Kiem HP, Slukvin I, Ryu BY, Sorrentino BP, Adair JE, Snyder SA, Compton AA, Torbett BE.

Blood. 2019 Aug 15. pii: blood.2019000040. doi: 10.1182/blood.2019000040. [Epub ahead of print]

PMID:
31416800
2.

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.

PMID:
31366580
3.

Autologous, Gene-Modified Hematopoietic Stem and Progenitor Cells Repopulate the Central Nervous System with Distinct Clonal Variants.

Peterson CW, Adair JE, Wohlfahrt ME, Deleage C, Radtke S, Rust B, Norman KK, Norgaard ZK, Schefter LE, Sghia-Hughes GM, Repetto A, Baldessari A, Murnane RD, Estes JD, Kiem HP.

Stem Cell Reports. 2019 Jul 9;13(1):91-104. doi: 10.1016/j.stemcr.2019.05.016. Epub 2019 Jun 13.

4.

TRAILshort Protects against CD4 T Cell Death during Acute HIV Infection.

Natesampillai S, Paim AC, Cummins NW, Chandrasekar AP, Bren GD, Lewin SR, Kiem HP, Badley AD.

J Immunol. 2019 Aug 1;203(3):718-724. doi: 10.4049/jimmunol.1900271. Epub 2019 Jun 12.

PMID:
31189571
5.

Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations.

Shahbazi R, Sghia-Hughes G, Reid JL, Kubek S, Haworth KG, Humbert O, Kiem HP, Adair JE.

Nat Mater. 2019 Oct;18(10):1124-1132. doi: 10.1038/s41563-019-0385-5. Epub 2019 May 27.

PMID:
31133730
6.

Lessons from London and Berlin: Designing A Scalable Gene Therapy Approach for HIV Cure.

Peterson CW, Kiem HP.

Cell Stem Cell. 2019 May 2;24(5):685-687. doi: 10.1016/j.stem.2019.04.010.

PMID:
31051132
7.

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells.

Radtke S, Perez AM, Venkataraman R, Reddy S, Haworth KG, Humbert O, Kiem HP, Peterson CW.

J Vis Exp. 2019 Feb 15;(144). doi: 10.3791/58933.

PMID:
30829324
8.

The response to lymphodepletion impacts PFS in patients with aggressive non-Hodgkin lymphoma treated with CD19 CAR T cells.

Hirayama AV, Gauthier J, Hay KA, Voutsinas JM, Wu Q, Gooley T, Li D, Cherian S, Chen X, Pender BS, Hawkins RM, Vakil A, Steinmetz RN, Acharya UH, Cassaday RD, Chapuis AG, Dhawale TM, Hendrie PC, Kiem HP, Lynch RC, Ramos J, Shadman M, Till BG, Riddell SR, Maloney DG, Turtle CJ.

Blood. 2019 Apr 25;133(17):1876-1887. doi: 10.1182/blood-2018-11-887067. Epub 2019 Feb 19.

PMID:
30782611
9.

Factors associated with durable EFS in adult B-cell ALL patients achieving MRD-negative CR after CD19 CAR T-cell therapy.

Hay KA, Gauthier J, Hirayama AV, Voutsinas JM, Wu Q, Li D, Gooley TA, Cherian S, Chen X, Pender BS, Hawkins RM, Vakil A, Steinmetz RN, Schoch G, Chapuis AG, Till BG, Kiem HP, Ramos JD, Shadman M, Cassaday RD, Acharya UH, Riddell SR, Maloney DG, Turtle CJ.

Blood. 2019 Apr 11;133(15):1652-1663. doi: 10.1182/blood-2018-11-883710. Epub 2019 Feb 6.

PMID:
30728140
10.

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction.

Lux CT, Pattabhi S, Berger M, Nourigat C, Flowers DA, Negre O, Humbert O, Yang JG, Lee C, Jacoby K, Bernstein I, Kiem HP, Scharenberg A, Rawlings DJ.

Mol Ther Methods Clin Dev. 2018 Dec 31;12:175-183. doi: 10.1016/j.omtm.2018.12.008. eCollection 2019 Mar 15.

11.

Human hematopoietic stem cell maintenance and myeloid cell development in next-generation humanized mouse models.

Sippel TR, Radtke S, Olsen TM, Kiem HP, Rongvaux A.

Blood Adv. 2019 Feb 12;3(3):268-274. doi: 10.1182/bloodadvances.2018023887. No abstract available.

12.

MISTRG mice support engraftment and assessment of nonhuman primate hematopoietic stem and progenitor cells.

Radtke S, Chan YY, Sippel TR, Kiem HP, Rongvaux A.

Exp Hematol. 2019 Feb;70:31-41.e1. doi: 10.1016/j.exphem.2018.12.003. Epub 2018 Dec 25.

13.

Long-Term Persistence of Anti-HIV Broadly Neutralizing Antibody-Secreting Hematopoietic Cells in Humanized Mice.

Kuhlmann AS, Haworth KG, Barber-Axthelm IM, Ironside C, Giese MA, Peterson CW, Kiem HP.

Mol Ther. 2019 Jan 2;27(1):164-177. doi: 10.1016/j.ymthe.2018.09.017. Epub 2018 Sep 27.

PMID:
30391142
14.

Evidence for persistence of the SHIV reservoir early after MHC haploidentical hematopoietic stem cell transplantation.

Colonna L, Peterson CW, Schell JB, Carlson JM, Tkachev V, Brown M, Yu A, Reddy S, Obenza WM, Nelson V, Polacino PS, Mack H, Hu SL, Zeleski K, Hoffman M, Olvera J, Furlan SN, Zheng H, Taraseviciute A, Hunt DJ, Betz K, Lane JF, Vogel K, Hotchkiss CE, Moats C, Baldessari A, Murnane RD, English C, Astley CA, Wangari S, Agricola B, Ahrens J, Iwayama N, May A, Stensland L, Huang MW, Jerome KR, Kiem HP, Kean LS.

Nat Commun. 2018 Oct 25;9(1):4438. doi: 10.1038/s41467-018-06736-7.

15.

Engineering resistance to CD33-targeted immunotherapy in normal hematopoiesis by CRISPR/Cas9-deletion of CD33 exon 2.

Humbert O, Laszlo GS, Sichel S, Ironside C, Haworth KG, Bates OM, Beddoe ME, Carrillo RR, Kiem HP, Walter RB.

Leukemia. 2019 Mar;33(3):762-808. doi: 10.1038/s41375-018-0277-8. Epub 2018 Oct 5. No abstract available.

PMID:
30291334
16.

Sorting Out the Best: Enriching Hematopoietic Stem Cells for Gene Therapy and Editing.

Radtke S, Humbert O, Kiem HP.

Mol Ther. 2018 Oct 3;26(10):2328-2329. doi: 10.1016/j.ymthe.2018.08.025. Epub 2018 Sep 13. No abstract available.

17.

Screening Clinical Cell Products for Replication Competent Retrovirus: The National Gene Vector Biorepository Experience.

Cornetta K, Duffy L, Feldman SA, Mackall CL, Davila ML, Curran KJ, Junghans RP, Tang JY, Kochenderfer JN, O'Cearbhaill R, Archer G, Kiem HP, Shah NN, Delbrook C, Kaplan R, Brentjens RJ, Rivière I, Sadelain M, Rosenberg SA.

Mol Ther Methods Clin Dev. 2018 Aug 17;10:371-378. doi: 10.1016/j.omtm.2018.08.006. eCollection 2018 Sep 21.

18.

Minimal conditioning in Fanconi anemia promotes multi-lineage marrow engraftment at 10-fold lower cell doses.

Haworth KG, Ironside C, Ramirez MA, Weitz S, Beard BC, Schwartz JD, Adair JE, Kiem HP.

J Gene Med. 2018 Oct;20(10-11):e3050. doi: 10.1002/jgm.3050. Epub 2018 Oct 1.

PMID:
30129972
19.

Efficient Enrichment of Gene-Modified Primary T Cells via CCR5-Targeted Integration of Mutant Dihydrofolate Reductase.

Paul B, Ibarra GSR, Hubbard N, Einhaus T, Astrakhan A, Rawlings DJ, Kiem HP, Peterson CW.

Mol Ther Methods Clin Dev. 2018 Apr 5;9:347-357. doi: 10.1016/j.omtm.2018.04.002. eCollection 2018 Jun 15.

20.

HIV infection results in clonal expansions containing integrations within pathogenesis-related biological pathways.

Haworth KG, Schefter LE, Norgaard ZK, Ironside C, Adair JE, Kiem HP.

JCI Insight. 2018 Jul 12;3(13). pii: 99127. doi: 10.1172/jci.insight.99127.

21.

Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A.

Adair JE, Chandrasekaran D, Sghia-Hughes G, Haworth KG, Woolfrey AE, Burroughs LM, Choi GY, Becker PS, Kiem HP.

Haematologica. 2018 Nov;103(11):1806-1814. doi: 10.3324/haematol.2018.194571. Epub 2018 Jul 5.

22.

Chimeric antigen receptor T-cell approaches to HIV cure.

Kuhlmann AS, Peterson CW, Kiem HP.

Curr Opin HIV AIDS. 2018 Sep;13(5):446-453. doi: 10.1097/COH.0000000000000485. Review.

PMID:
29878913
23.

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.

24.

Next-Generation Conditioning for Bone Marrow Transplantation: Paving the Way for CAR-T Cell-Based Conditioning.

Haworth KG, Kiem HP.

Mol Ther. 2018 May 2;26(5):1167-1168. doi: 10.1016/j.ymthe.2018.04.011. No abstract available.

25.

Differential impact of transplantation on peripheral and tissue-associated viral reservoirs: Implications for HIV gene therapy.

Peterson CW, Wang J, Deleage C, Reddy S, Kaur J, Polacino P, Reik A, Huang ML, Jerome KR, Hu SL, Holmes MC, Estes JD, Kiem HP.

PLoS Pathog. 2018 Apr 19;14(4):e1006956. doi: 10.1371/journal.ppat.1006956. eCollection 2018 Apr.

26.

Corrigendum: Suppression of luteinizing hormone enhances HSC recovery after hematopoietic injury.

Velardi E, Tsai JJ, Radtke S, Cooper K, Argyropoulos KV, Jae-Hung S, Young LF, Lazrak A, Smith OM, Lieberman S, Kreines F, Shono Y, Wertheimer T, Jenq RR, Hanash AM, Narayan P, Lei Z, Moore MA, Kiem HP, van den Brink MRM, Dudakov JA.

Nat Med. 2018 Apr 10;24(4):525. doi: 10.1038/nm0418-525b.

PMID:
29634695
27.

Correction: Long-term persistence and function of hematopoietic stem cell-derived chimeric antigen receptor T cells in a nonhuman primate model of HIV/AIDS.

Zhen A, Peterson CW, Carrillo MA, Reddy SS, Youn CS, Lam BB, Chang NY, Martin HA, Rick JW, Kim J, Neel NC, Rezek VK, Kamata M, Chen ISY, Zack JA, Kiem HP, Kitchen SG.

PLoS Pathog. 2018 Mar 12;14(3):e1006891. doi: 10.1371/journal.ppat.1006891. eCollection 2018 Mar.

28.

Suppression of luteinizing hormone enhances HSC recovery after hematopoietic injury.

Velardi E, Tsai JJ, Radtke S, Cooper K, Argyropoulos KV, Jae-Hung S, Young LF, Lazrak A, Smith OM, Lieberman S, Kreines F, Shono Y, Wertheimer T, Jenq RR, Hanash AM, Narayan P, Lei Z, Moore MA, Kiem HP, van den Brink MRM, Dudakov JA.

Nat Med. 2018 Feb;24(2):239-246. doi: 10.1038/nm.4470. Epub 2018 Jan 8. Erratum in: Nat Med. 2018 Apr 10;24(4):525.

29.

Long-term persistence and function of hematopoietic stem cell-derived chimeric antigen receptor T cells in a nonhuman primate model of HIV/AIDS.

Zhen A, Peterson CW, Carrillo MA, Reddy SS, Youn CS, Lam BB, Chang NY, Martin HA, Rick JW, Kim J, Neel NC, Rezek VK, Kamata M, Chen ISY, Zack JA, Kiem HP, Kitchen SG.

PLoS Pathog. 2017 Dec 28;13(12):e1006753. doi: 10.1371/journal.ppat.1006753. eCollection 2017 Dec. Erratum in: PLoS Pathog. 2018 Mar 12;14 (3):e1006891.

30.

A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies.

Humbert O, Peterson CW, Norgaard ZK, Radtke S, Kiem HP.

Mol Ther Methods Clin Dev. 2017 Nov 21;8:75-86. doi: 10.1016/j.omtm.2017.11.005. eCollection 2018 Mar 16.

31.

Cell and Gene Therapy for HIV Cure.

Peterson CW, Kiem HP.

Curr Top Microbiol Immunol. 2018;417:211-248. doi: 10.1007/82_2017_71. Review.

PMID:
29256135
32.

A Combined In Vivo HSC Transduction/Selection Approach Results in Efficient and Stable Gene Expression in Peripheral Blood Cells in Mice.

Wang H, Richter M, Psatha N, Li C, Kim J, Liu J, Ehrhardt A, Nilsson SK, Cao B, Palmer D, Ng P, Izsvák Z, Haworth KG, Kiem HP, Papayannopoulou T, Lieber A.

Mol Ther Methods Clin Dev. 2017 Nov 10;8:52-64. doi: 10.1016/j.omtm.2017.11.004. eCollection 2018 Mar 16.

33.

A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates.

Radtke S, Adair JE, Giese MA, Chan YY, Norgaard ZK, Enstrom M, Haworth KG, Schefter LE, Kiem HP.

Sci Transl Med. 2017 Nov 1;9(414). pii: eaan1145. doi: 10.1126/scitranslmed.aan1145.

34.

Hematopoietic Stem Cell Approaches to Cancer.

Adair JE, Kubek SP, Kiem HP.

Hematol Oncol Clin North Am. 2017 Oct;31(5):897-912. doi: 10.1016/j.hoc.2017.06.012. Review.

PMID:
28895855
35.

In Vivo Hematopoietic Stem Cell Transduction.

Richter M, Stone D, Miao C, Humbert O, Kiem HP, Papayannopoulou T, Lieber A.

Hematol Oncol Clin North Am. 2017 Oct;31(5):771-785. doi: 10.1016/j.hoc.2017.06.001. Review.

36.

Nanoparticle Biokinetics in Mice and Nonhuman Primates.

Chiarelli PA, Revia RA, Stephen ZR, Wang K, Jeon M, Nelson V, Kievit FM, Sham J, Ellenbogen RG, Kiem HP, Zhang M.

ACS Nano. 2017 Sep 26;11(9):9514-9524. doi: 10.1021/acsnano.7b05377. Epub 2017 Sep 18.

37.

Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers.

Moffett HF, Coon ME, Radtke S, Stephan SB, McKnight L, Lambert A, Stoddard BL, Kiem HP, Stephan MT.

Nat Commun. 2017 Aug 30;8(1):389. doi: 10.1038/s41467-017-00505-8.

38.

CCR5-edited gene therapies for HIV cure: Closing the door to viral entry.

Haworth KG, Peterson CW, Kiem HP.

Cytotherapy. 2017 Nov;19(11):1325-1338. doi: 10.1016/j.jcyt.2017.05.013. Epub 2017 Jul 24. Review.

PMID:
28751153
39.

In Vivo Murine-Matured Human CD3+ Cells as a Preclinical Model for T Cell-Based Immunotherapies.

Haworth KG, Ironside C, Norgaard ZK, Obenza WM, Adair JE, Kiem HP.

Mol Ther Methods Clin Dev. 2017 May 17;6:17-30. doi: 10.1016/j.omtm.2017.05.004. eCollection 2017 Sep 15.

40.

Refining Current Scientific Priorities and Identifying New Scientific Gaps in HIV-Related Heart, Lung, Blood, and Sleep Research.

Twigg HL 3rd, Crystal R, Currier J, Ridker P, Berliner N, Kiem HP, Rutherford G, Zou S, Glynn S, Wong R, Peprah E, Engelgau M, Creazzo T, Colombini-Hatch S, Caler E.

AIDS Res Hum Retroviruses. 2017 Sep;33(9):889-897. doi: 10.1089/AID.2017.0026. Epub 2017 Jun 26.

41.

Autologous Stem Cell Transplantation Disrupts Adaptive Immune Responses during Rebound Simian/Human Immunodeficiency Virus Viremia.

Reeves DB, Peterson CW, Kiem HP, Schiffer JT.

J Virol. 2017 Jun 9;91(13). pii: e00095-17. doi: 10.1128/JVI.00095-17. Print 2017 Jul 1.

42.

Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.

Singh S, Khan I, Khim S, Seymour B, Sommer K, Wielgosz M, Norgaard Z, Kiem HP, Adair J, Liggitt D, Nienhuis A, Rawlings DJ.

Mol Ther Methods Clin Dev. 2016 Dec 18;4:1-16. doi: 10.1016/j.omtm.2016.11.001. eCollection 2017 Mar 17.

43.

Endothelial Cells Promote Expansion of Long-Term Engrafting Marrow Hematopoietic Stem and Progenitor Cells in Primates.

Gori JL, Butler JM, Kunar B, Poulos MG, Ginsberg M, Nolan DJ, Norgaard ZK, Adair JE, Rafii S, Kiem HP.

Stem Cells Transl Med. 2017 Mar;6(3):864-876. doi: 10.5966/sctm.2016-0240. Epub 2016 Oct 14.

44.

Loss of immune homeostasis dictates SHIV rebound after stem-cell transplantation.

Peterson CW, Benne C, Polacino P, Kaur J, McAllister CE, Filali-Mouhim A, Obenza W, Pecor TA, Huang ML, Baldessari A, Murnane RD, Woolfrey AE, Jerome KR, Hu SL, Klatt NR, DeRosa S, Sékaly RP, Kiem HP.

JCI Insight. 2017 Feb 23;2(4):e91230. doi: 10.1172/jci.insight.91230.

45.

Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia.

Adair JE, Sevilla J, Heredia CD, Becker PS, Kiem HP, Bueren J.

Curr Gene Ther. 2017;16(5):338-348. doi: 10.2174/1566523217666170119113029. Review.

PMID:
28103787
46.

Virion Structure of Black Queen Cell Virus, a Common Honeybee Pathogen.

Spurny R, Přidal A, Pálková L, Kiem HKT, de Miranda JR, Plevka P.

J Virol. 2017 Feb 28;91(6). pii: e02100-16. doi: 10.1128/JVI.02100-16. Print 2017 Mar 15.

47.

Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia.

Thakar MS, Bonfim C, Walters MC, Storb R, Pasquini R, Burroughs L, Sandmaier BM, Woolfrey A, Kiem HP.

Bone Marrow Transplant. 2017 Apr;52(4):570-573. doi: 10.1038/bmt.2016.301. Epub 2017 Jan 9.

48.

Devouring the Hematopoietic Stem Cell: Setting the Table for Marrow Cell Transplantation.

Cowan MJ, Kiem HP.

Mol Ther. 2016 Nov;24(11):1892-1894. doi: 10.1038/mt.2016.193. No abstract available.

49.

Cell-Delivered Entry Inhibitors for HIV-1: CCR5 Downregulation and Blocking Virus/Membrane Fusion in Defending the Host Cell Population.

Symonds G, Bartlett JS, Kiem HP, Tsie M, Breton L.

AIDS Patient Care STDS. 2016 Dec;30(12):545-550.

PMID:
27905841
50.

Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia.

Bonfim C, Ribeiro L, Nichele S, Loth G, Bitencourt M, Koliski A, Kuwahara C, Fabro AL, Pereira NF, Pilonetto D, Thakar M, Kiem HP, Page K, Fuchs EJ, Eapen M, Pasquini R.

Biol Blood Marrow Transplant. 2017 Feb;23(2):310-317. doi: 10.1016/j.bbmt.2016.11.006. Epub 2016 Nov 7.

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