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HIVFanconi AnemiaStem Cell Biology and TransplantationNanoparticle StudiesVirus Studies


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. ”Autologous, Gene-Modified Hematopoietic Stem and Progenitor Cells Repopulate the Central Nervous System with Distinct Clonal Variants.” Stem Cell Reports. 2019 Jul 9;13(1):91-104. doi: 10.1016/j.stemcr.2019.05.016. Epub 2019 Jun 13.

Peterson CW, Kiem HP. “Lessons from London and Berlin: Designing A Scalable Gene Therapy Approach for HIV Cure. Cell Stem Cell.” 2019 May 2;24(5):685-687. doi: 10.1016/j.stem.2019.04.010.

Kuhlmann,A. S., K. G. Haworth, I. M. Barber-Axthelm, C. Ironside, M. A. Giese, C. W. Peterson, and H.P. Kiem. "Long-Term Persistence of Anti-HIV Broadly Neutralizing Antibody-Secreting Hematopoietic Cells in Humanized Mice." Mol Ther 27.1 (2019): 164-77. Print.

Haworth, K. G., L. E. Schefter, Z. K. Norgaard, C. Ironside, J. E. Adair, and H.P. Kiem. "HIV Infection Results in Clonal Expansions Containing Integrations within Pathogenesis-Related Biological Pathways." JCI Insight 3.13 (2018). Print.

Kuhlmann, A. S., C. W. Peterson, and H.P. Kiem. "Chimeric Antigen Receptor T-Cell Approaches to HIV Cure." Curr Opin HIV AIDS 13.5 (2018): 446-53. Print

Zhen, A., C. W. Peterson, M. A. Carrillo, S. S. Reddy, C. S. Youn, B. B. Lam, N. Y. Chang, H. A. Martin, J. W. Rick, J. Kim, N. C. Neel, V. K. Rezek, M. Kamata, I. S. Y. Chen, J. A. Zack, H.P. Kiem, and S. G. Kitchen. "Correction: Long-Term Persistence and Function of Hematopoietic Stem Cell-Derived Chimeric Antigen Receptor T Cells in a Nonhuman Primate Model of HIV/Aids." PLoS Pathog 14.3 (2018): e1006891. Print.

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.

Haworth, K. G., C. W. Peterson, and H.P. Kiem. "Ccr5-Edited Gene Therapies for HIV Cure: Closing the Door to Viral Entry." Cytotherapy 19.11 (2017): 1325-38. Print.

Kuhlmann AS, KG Haworth, IM Barber-Axthelm, C Ironside, MA Giese, Peterson CW, H.P. Kiem. "Long-Term Persistence and Function of Hematopoietic Stem Cell-Derived Chimeric Antigen Receptor T Cells in a Nonhuman Primate Model of HIV/Aids." PLoS Pathog 13.12 (2017): e1006753. Print.

Twigg, H. L., 3rd, R. Crystal, J. Currier, P. Ridker, N. Berliner, H.P. Kiem, G. Rutherford, S. Zou, S. Glynn, R. Wong, E. Peprah, M. Engelgau, T. Creazzo, S. Colombini-Hatch, and E. Caler. "Refining Current Scientific Priorities and Identifying New Scientific Gaps in HIV-Related Heart, Lung, Blood, and Sleep Research." AIDS Res Hum Retroviruses 33.9 (2017): 889-97. Print.

Deeks, S. G., S. R. Lewin, A. L. Ross, J. Ananworanich, M. Benkirane, P. Cannon, N. Chomont, D. Douek, J. D. Lifson, Y. R. Lo, D. Kuritzkes, D. Margolis, J. Mellors, D. Persaud, J. D. Tucker, F. Barre-Sinoussi, G. Alter, J. Auerbach, B. Autran, D. H. Barouch, G. Behrens, M. Cavazzana, Z. Chen, E. A. Cohen, G. M. Corbelli, S. Eholie, N. Eyal, S. Fidler, L. Garcia, C. Grossman, G. Henderson, T. J. Henrich, R. Jefferys, H.P. Kiem, J. McCune, K. Moodley, P. A. Newman, M. Nijhuis, M. S. Nsubuga, M. Ott, S. Palmer, D. Richman, A. Saez-Cirion, M. Sharp, J. Siliciano, G. Silvestri, J. Singh, B. Spire, J. Taylor, M. Tolstrup, S. Valente, J. van Lunzen, R. Walensky, I. Wilson, and J. Zack. "International Aids Society Global Scientific Strategy: Towards an HIV Cure 2016." Nat Med 22.8 (2016): 839-50. Print.

Dimitrov, D. T., H.P. Kiem, K. R. Jerome, C. Johnston, and J. T. Schiffer. "A Curative Regimen Would Decrease HIV Prevalence but Not HIV Incidence Unless Targeted to an Art-Naive Population." Sci Rep 6 (2016): 22183. Print.

Lederman, M. M., P. M. Cannon, J. S. Currier, C. H. June, H.P. Kiem, D. R. Kuritzkes, S. R. Lewin, D. M. Margolis, J. M. McCune, J. W. Mellors, T. W. Schacker, R. P. Sekaly, P. Tebas, B. D. Walker, and D. C. Douek. "A Cure for HIV Infection: "Not in My Lifetime" or "Just around the Corner"?" Pathog Immun 1.1 (2016): 154-64. Print.

Symonds, G., J. S. Bartlett, H.P. Kiem, M. Tsie, and L. Breton. "Cell-Delivered Entry Inhibitors for Hiv-1: Ccr5 Downregulation and Blocking Virus/Membrane Fusion in Defending the Host Cell Population." AIDS Patient Care STDS 30.12 (2016): 545-50. Print.

Fanconi Anemia

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. “Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes.” Blood. 2019 Oct 17;134(16):1298-1311. doi: 10.1182/blood.2019000040.

Peterson, C. W., and H.P. Kiem. "Cell and Gene Therapy for HIV Cure." Curr Top Microbiol Immunol 417 (2018): 211-48. Print.

Adair, J. E., D. Chandrasekaran, G. Sghia-Hughes, K. G. Haworth, A. E. Woolfrey, L. M. Burroughs, G. Y. Choi, P. S. Becker, and H.P. Kiem. "Novel Lineage Depletion Preserves Autologous Blood Stem Cells for Gene Therapy of Fanconi Anemia Complementation Group A." Haematologica 103.11 (2018): 1806-14. Print.

Haworth, K. G., C. Ironside, M. A. Ramirez, S. Weitz, B. C. Beard, J. D. Schwartz, J. E. Adair, and H.P. Kiem. "Minimal Conditioning in Fanconi Anemia Promotes Multi-Lineage Marrow Engraftment at 10-Fold Lower Cell Doses." J Gene Med 20.10-11 (2018): e3050. Print.

Adair, J. E., J. Sevilla, C. D. Heredia, P. S. Becker, H.P. Kiem, and J. Bueren. "Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia." Curr Gene Ther 16.5 (2017): 338-48. Print.

Bonfim, C., L. Ribeiro, S. Nichele, G. Loth, M. Bitencourt, A. Koliski, C. Kuwahara, A. L. Fabro, N. F. Pereira, D. Pilonetto, M. Thakar, H.P. Kiem, K. Page, E. J. Fuchs, M. Eapen, and R. Pasquini. "Haploidentical Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Children and Adolescents with Fanconi Anemia." Biol Blood Marrow Transplant 23.2 (2017): 310-17. Print.

Reeves, D. B., C. W. Peterson, H.P. Kiem, and J. T. Schiffer. "Autologous Stem Cell Transplantation Disrupts Adaptive Immune Responses During Rebound Simian/Human Immunodeficiency Virus Viremia." J Virol 91.13 (2017). Print.

Romano Ibarra, G. S., B. Paul, B. D. Sather, P. M. Younan, K. Sommer, J. P. Kowalski, M. Hale, B. Stoddard, J. Jarjour, A. Astrakhan, H.P. Kiem, and D. J. Rawlings. "Efficient Modification of the Ccr5 Locus in Primary Human T Cells with Megatal Nuclease Establishes Hiv-1 Resistance." Mol Ther Nucleic Acids 5.8 (2016): e352. Print.

Stem Cell Biology and Transplantation

Rajawat YS, Humbert O, Kiem HP. “In-Vivo Gene Therapy with Foamy Virus Vectors.” Viruses. 2019 Nov 23;11(12). pii: E1091. doi: 10.3390/v11121091. Review. PMID: 31771194 

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. “Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.” Sci Transl Med. 2019 Jul 31;11(503). pii: eaaw3768. doi: 10.1126/scitranslmed.aaw3768.

Radtke, S., Y. Y. Chan, T. R. Sippel, H.P. Kiem, and A. Rongvaux. "Mistrg Mice Support Engraftment and Assessment of Nonhuman Primate Hematopoietic Stem and Progenitor Cells." Exp Hematol 70 (2019): 31-41.e1. Print.

Lux, C. T., S. Pattabhi, M. Berger, C. Nourigat, D. A. Flowers, O. Negre, O. Humbert, J. G. Yang, C. Lee, K. Jacoby, I. Bernstein, H.P. Kiem, A. Scharenberg, and D. J. Rawlings. "Talen-Mediated Gene Editing of Hbg in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction." Mol Ther Methods Clin Dev 12 (2019): 175-83. Print.

Hay, K. A., J. Gauthier, A. V. Hirayama, J. M. Voutsinas, Q. Wu, D. Li, T. A. Gooley, S. Cherian, X. Chen, B. S. Pender, R. M. Hawkins, A. Vakil, R. N. Steinmetz, G. Schoch, A. G. Chapuis, B. G. Till, H.P. Kiem, J. D. Ramos, M. Shadman, R. D. Cassaday, U. H. Acharya, S. R. Riddell, D. G. Maloney, and C. J. Turtle. "Factors Associated with Durable Efs in Adult B-Cell All Patients Achieving Mrd-Negative Cr after Cd19 Car T-Cell Therapy." Blood 133.15 (2019): 1652-63. Print.

Hirayama, A. V., J. Gauthier, K. A. Hay, J. M. Voutsinas, Q. Wu, T. Gooley, D. Li, S. Cherian, X. Chen, B. S. Pender, R. M. Hawkins, A. Vakil, R. N. Steinmetz, U. H. Acharya, R. D. Cassaday, A. G. Chapuis, T. M. Dhawale, P. C. Hendrie, H.P. Kiem, R. C. Lynch, J. Ramos, M. Shadman, B. G. Till, S. R. Riddell, D. G. Maloney, and C. J. Turtle. "The Response to Lymphodepletion Impacts Pfs in Patients with Aggressive Non-Hodgkin Lymphoma Treated with Cd19 Car T Cells." Blood 133.17 (2019): 1876-87. Print.

Humbert, O., G. S. Laszlo, S. Sichel, C. Ironside, K. G. Haworth, O. M. Bates, M. E. Beddoe, R. R. Carrillo, H.P. Kiem, and R. B. Walter. "Engineering Resistance to Cd33-Targeted Immunotherapy in Normal Hematopoiesis by Crispr/Cas9-Deletion of Cd33 Exon 2." Leukemia 33.3 (2019): 762-808. Print.

Sippel, T. R., S. Radtke, T. M. Olsen, H.P. Kiem, and A. Rongvaux. "Human Hematopoietic Stem Cell Maintenance and Myeloid Cell Development in Next-Generation Humanized Mouse Models." Blood Adv 3.3 (2019): 268-74. Print.

Radtke, S., A. M. Perez, R. Venkataraman, S. Reddy, K. G. Haworth, O. Humbert, H.P. Kiem, and C. W. Peterson. "Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells." J Vis Exp.144 (2019). Print.

Colonna, L., C. W. Peterson, J. B. Schell, J. M. Carlson, V. Tkachev, M. Brown, A. Yu, S. Reddy, W. M. Obenza, V. Nelson, P. S. Polacino, H. Mack, S. L. Hu, K. Zeleski, M. Hoffman, J. Olvera, S. N. Furlan, H. Zheng, A. Taraseviciute, D. J. Hunt, K. Betz, J. F. Lane, K. Vogel, C. E. Hotchkiss, C. Moats, A. Baldessari, R. D. Murnane, C. English, C. A. Astley, S. Wangari, B. Agricola, J. Ahrens, N. Iwayama, A. May, L. Stensland, M. W. Huang, K. R. Jerome, H.P. Kiem, and L. S. Kean. "Evidence for Persistence of the Shiv Reservoir Early after Mhc Haploidentical Hematopoietic Stem Cell Transplantation." Nat Commun 9.1 (2018): 4438. Print.

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. "Suppression of Luteinizing Hormone Enhances Hsc Recovery after Hematopoietic Injury." 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.

Humbert, O., F. Chan, Y. S. Rajawat, T. R. Torgerson, C. R. Burtner, N. W. Hubbard, D. Humphrys, Z. K. Norgaard, P. O'Donnell, J. E. Adair, G. D. Trobridge, A. M. Scharenberg, P. J. Felsburg, D. J. Rawlings, and H.P. Kiem. "Rapid Immune Reconstitution of Scid-X1 Canines after G-Csf/Amd3100 Mobilization and in Vivo Gene Therapy." Blood Adv 2.9 (2018): 987-99. Print.

Radtke, S., O. Humbert, and H.P. Kiem. "Sorting out the Best: Enriching Hematopoietic Stem Cells for Gene Therapy and Editing." Mol Ther 26.10 (2018): 2328-29. Print.

Haworth, K. G., and H.P. Kiem. "Next-Generation Conditioning for Bone Marrow Transplantation: Paving the Way for Car-T Cell-Based Conditioning." Mol Ther 26.5 (2018): 1167-68. Print.

Paul, B., G. S. R. Ibarra, N. Hubbard, T. Einhaus, A. Astrakhan, D. J. Rawlings, H.P. Kiem, and C. W. Peterson. "Efficient Enrichment of Gene-Modified Primary T Cells Via Ccr5-Targeted Integration of Mutant Dihydrofolate Reductase." Mol Ther Methods Clin Dev 9 (2018): 347-57. Print.

Humbert, O., C. W. Peterson, Z. K. Norgaard, S. Radtke, and H.P. Kiem. "A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for Beta-Hemoglobinopathies." Mol Ther Methods Clin Dev 8 (2018): 75-86. Print.

Wang, H., M. Richter, N. Psatha, C. Li, J. Kim, J. Liu, A. Ehrhardt, S. K. Nilsson, B. Cao, D. Palmer, P. Ng, Z. Izsvak, K. G. Haworth, H.P. Kiem, T. Papayannopoulou, and A. Lieber. "A Combined in Vivo Hsc Transduction/Selection Approach Results in Efficient and Stable Gene Expression in Peripheral Blood Cells in Mice." Mol Ther Methods Clin Dev 8 (2018): 52-64. Print.

Peterson, C. W., J. Wang, C. Deleage, S. Reddy, J. Kaur, P. Polacino, A. Reik, M. L. Huang, K. R. Jerome, S. L. Hu, M. C. Holmes, J. D. Estes, and H.P. Kiem. "Differential Impact of Transplantation on Peripheral and Tissue-Associated Viral Reservoirs: Implications for HIV Gene Therapy." PLoS Pathog 14.4 (2018): e1006956. Print.

Adair, J. E., S. P. Kubek, and H.P. Kiem. "Hematopoietic Stem Cell Approaches to Cancer." Hematol Oncol Clin North Am 31.5 (2017): 897-912. Print.

Gori, J. L., J. M. Butler, B. Kunar, M. G. Poulos, M. Ginsberg, D. J. Nolan, Z. K. Norgaard, J. E. Adair, S. Rafii, and H.P. Kiem. "Endothelial Cells Promote Expansion of Long-Term Engrafting Marrow Hematopoietic Stem and Progenitor Cells in Primates." Stem Cells Transl Med 6.3 (2017): 864-76. Print

Haworth, K. G., C. Ironside, Z. K. Norgaard, W. M. Obenza, J. E. Adair, and H.P. Kiem. "In Vivo Murine-Matured Human Cd3(+) Cells as a Preclinical Model for T Cell-Based Immunotherapies." Mol Ther Methods Clin Dev 6 (2017): 17-30. Print.

Radtke, S., J. E. Adair, M. A. Giese, Y. Y. Chan, Z. K. Norgaard, M. Enstrom, K. G. Haworth, L. E. Schefter, and H.P. Kiem. "A Distinct Hematopoietic Stem Cell Population for Rapid Multilineage Engraftment in Nonhuman Primates." Sci Transl Med 9.414 (2017). Print.

Peterson, C. W., C. Benne, P. Polacino, J. Kaur, C. E. McAllister, A. Filali-Mouhim, W. Obenza, T. A. Pecor, M. L. Huang, A. Baldessari, R. D. Murnane, A. E. Woolfrey, K. R. Jerome, S. L. Hu, N. R. Klatt, S. DeRosa, R. P. Sekaly, and H.P. Kiem. "Loss of Immune Homeostasis Dictates Shiv Rebound after Stem-Cell Transplantation." JCI Insight 2.4 (2017): e91230. Print.

Richter, M., D. Stone, C. Miao, O. Humbert, H.P. Kiem, T. Papayannopoulou, and A. Lieber. "In Vivo Hematopoietic Stem Cell Transduction." Hematol Oncol Clin North Am 31.5 (2017): 771-85. Print.

Singh, S., I. Khan, S. Khim, B. Seymour, K. Sommer, M. Wielgosz, Z. Norgaard, H.P. Kiem, J. Adair, D. Liggitt, A. Nienhuis, and D. J. Rawlings. "Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector." Mol Ther Methods Clin Dev 4 (2017): 1-16. Print.

Adair, J. E., T. Waters, K. G. Haworth, S. P. Kubek, G. D. Trobridge, J. D. Hocum, S. Heimfeld, and H.P. Kiem. "Semi-Automated Closed System Manufacturing of Lentivirus Gene-Modified Haematopoietic Stem Cells for Gene Therapy." Nat Commun 7 (2016): 13173. Print.

Jenkins, C. L., M. L. Bagarazzi, H.P. Kiem, A. J. Levine, D. Ando, C. H. June, T. Reid, and D. W. Emery. "Rethinking the Regulatory Infrastructure for Human Gene Transfer Clinical Trials." Mol Ther 24.7 (2016): 1173-7. Print.

Peterson, C. W., K. G. Haworth, B. P. Burke, P. Polacino, K. K. Norman, J. E. Adair, S. L. Hu, J. S. Bartlett, G. P. Symonds, and H.P. Kiem. "Multilineage Polyclonal Engraftment of Cal-1 Gene-Modified Cells and in Vivo Selection after Shiv Infection in a Nonhuman Primate Model of Aids." Mol Ther Methods Clin Dev 3 (2016): 16007. Print.

Peterson, C. W., J. Wang, K. K. Norman, Z. K. Norgaard, O. Humbert, C. K. Tse, J. J. Yan, R. G. Trimble, D. A. Shivak, E. J. Rebar, P. D. Gregory, M. C. Holmes, and H.P. Kiem. "Long-Term Multilineage Engraftment of Autologous Genome-Edited Hematopoietic Stem Cells in Nonhuman Primates." Blood 127.20 (2016): 2416-26. Print.

Johnston, C., R. Harrington, R. Jain, J. Schiffer, H.P. Kiem, and A. Woolfrey. "Safety and Efficacy of Combination Antiretroviral Therapy in Human Immunodeficiency Virus-Infected Adults Undergoing Autologous or Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancies." Biol Blood Marrow Transplant 22.1 (2016): 149-56. Print.

Cowan, M. J., and H.P. Kiem. "Devouring the Hematopoietic Stem Cell: Setting the Table for Marrow Cell Transplantation." Mol Ther 24.11 (2016): 1892-94. Print.

Radtke, S., K. G. Haworth, and H.P. Kiem. "The Frequency of Multipotent Cd133(+)Cd45ra(-)Cd34(+) Hematopoietic Stem Cells Is Not Increased in Fetal Liver Compared with Adult Stem Cell Sources." Exp Hematol 44.6 (2016): 502-7. Print.

Richter, M., K. Saydaminova, R. Yumul, R. Krishnan, J. Liu, E. E. Nagy, M. Singh, Z. Izsvak, R. Cattaneo, W. Uckert, D. Palmer, P. Ng, K. G. Haworth, H.P. Kiem, A. Ehrhardt, T. Papayannopoulou, and A. Lieber. "In Vivo Transduction of Primitive Mobilized Hematopoietic Stem Cells after Intravenous Injection of Integrating Adenovirus Vectors." Blood 128.18 (2016): 2206-17. Print.

Burtner, C. R., D. Chandrasekaran, E. B. Santos, B. C. Beard, J. E. Adair, D. K. Hamlin, D. S. Wilbur, B. M. Sandmaier, and H.P. Kiem. "(211)Astatine-Conjugated Monoclonal Cd45 Antibody-Based Nonmyeloablative Conditioning for Stem Cell Gene Therapy." Hum Gene Ther 26.6 (2015): 399-406. Print.

Egerer, L., H.P. Kiem, and D. von Laer. "C Peptides as Entry Inhibitors for Gene Therapy." Adv Exp Med Biol 848 (2015): 191-209. Print.

Felsburg, P. J., S. S. De Ravin, H. L. Malech, B. P. Sorrentino, C. Burtner, and H.P. Kiem. "Gene Therapy Studies in a Canine Model of X-Linked Severe Combined Immunodeficiency." Hum Gene Ther Clin Dev 26.1 (2015): 50-6. Print

Peterson, C. W., K. G. Haworth, P. Polacino, M. L. Huang, C. Sykes, W. M. Obenza, A. C. Repetto, A. Kashuba, R. Bumgarner, S. C. DeRosa, A. E. Woolfrey, K. R. Jerome, J. I. Mullins, S. L. Hu, and H.P. Kiem. "Lack of Viral Control and Development of Combination Antiretroviral Therapy Escape Mutations in Macaques after Bone Marrow Transplantation." Aids 29.13 (2015): 1597-606. Print.

Chandrasekaran, D., B. Nakamoto, K. L. Watts, H.P. Kiem, and T. Papayannopoulou. "Modeling Promising Nonmyeloablative Conditioning Regimens in Nonhuman Primates." Hum Gene Ther 25.12 (2014): 1013-22. Print.

Fares, I., J. Chagraoui, Y. Gareau, S. Gingras, R. Ruel, N. Mayotte, E. Csaszar, D. J. Knapp, P. Miller, M. Ngom, S. Imren, D. C. Roy, K. L. Watts, H.P. Kiem, R. Herrington, N. N. Iscove, R. K. Humphries, C. J. Eaves, S. Cohen, A. Marinier, P. W. Zandstra, and G. Sauvageau. "Cord Blood Expansion. Pyrimidoindole Derivatives Are Agonists of Human Hematopoietic Stem Cell Self-Renewal." Science 345.6203 (2014): 1509-12. Print.

Kiem, H.P., P. I. Arumugam, C. R. Burtner, C. F. Fox, B. C. Beard, P. Dexheimer, J. E. Adair, and P. Malik. "Pigtailed Macaques as a Model to Study Long-Term Safety of Lentivirus Vector-Mediated Gene Therapy for Hemoglobinopathies." Mol Ther Methods Clin Dev 1 (2014): 14055. Print.

Kiem, H.P., C. Baum, F. D. Bushman, B. J. Byrne, B. J. Carter, J. Cavagnaro, H. L. Malech, J. R. Mendell, L. M. Naldini, B. P. Sorrentino, D. A. Williams, and T. R. Flotte. "Charting a Clear Path: The Asgct Standardized Pathways Conference." Mol Ther 22.7 (2014): 1235-38. Print.

Wang, C. X., B. D. Sather, X. Wang, J. Adair, I. Khan, S. Singh, S. Lang, A. Adams, G. Curinga, H.P. Kiem, C. H. Miao, D. J. Rawlings, and B. E. Torbett. "Rapamycin Relieves Lentiviral Vector Transduction Resistance in Human and Mouse Hematopoietic Stem Cells." Blood 124.6 (2014): 913-23. Print.

Nanoparticle Studies

Shahbazi R, Sghia-Hughes G, Reid JL, Kubek S, Haworth KG, Humbert O, Kiem HP, Adair JE. “Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations.” Nat Mater. 2019 Oct;18(10):1124-1132. doi: 10.1038/s41563-019-0385-5. Epub 2019 May 27.

Chiarelli, P. A., R. A. Revia, Z. R. Stephen, K. Wang, M. Jeon, V. Nelson, F. M. Kievit, J. Sham, R. G. Ellenbogen, H.P. Kiem, and M. Zhang. "Nanoparticle Biokinetics in Mice and Nonhuman Primates." ACS Nano 11.9 (2017): 9514-24. Print.

Virus Studies

Cornetta, K., L. Duffy, S. A. Feldman, C. L. Mackall, M. L. Davila, K. J. Curran, R. P. Junghans, J. Y. Tang, J. N. Kochenderfer, R. O'Cearbhaill, G. Archer, H.P. Kiem, N. N. Shah, C. Delbrook, R. Kaplan, R. J. Brentjens, I. Riviere, M. Sadelain, and S. A. Rosenberg. "Screening Clinical Cell Products for Replication Competent Retrovirus: The National Gene Vector Biorepository Experience." Mol Ther Methods Clin Dev 10 (2018): 371-78. Print.

Humbert, O., D. W. Gisch, M. E. Wohlfahrt, A. B. Adams, P. D. Greenberg, T. M. Schmitt, G. D. Trobridge, and H.P. Kiem. "Development of Third-Generation Cocal Envelope Producer Cell Lines for Robust Lentiviral Gene Transfer into Hematopoietic Stem Cells and T-Cells." Mol Ther 24.7 (2016): 1237-46. Print

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