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456  Part V:  Therapeutic Principles  Chapter 30:  Regenerative Medicine: Multipotential Cell Therapy for Tissue Repair   457




                    151. Kroon E, Martinson LA, Kadoya K, et al: Pancreatic endoderm derived from human     169. Goessling W, Allen RS, Guan X, et al: Prostaglandin E2 enhances human cord blood
                     embryonic stem cells generates glucose-responsive insulin-secreting cells in vivo. Nat   stem cell xenotransplants and shows long-term safety in preclinical nonhuman primate
                     Biotechnol 26(4):443–452, 2008.                       transplant models. Cell Stem Cell 8(4):445–458, 2011.
                    152. Rezania A, Bruin JE, Riedel MJ, et al: Maturation of human embryonic stem cell-     170. Perez EE, Wang J, Miller JC, et al: Establishment of HIV-1 resistance in CD4+ T cells by
                     derived pancreatic progenitors into functional islets capable of treating pre-existing   genome editing using zinc-finger nucleases. Nat Biotechnol 26(7):808–816, 2008.
                     diabetes in mice. Diabetes 61(8):2016–2029, 2012.    171. McElrath MJ, Smythe K, Randolph-Habecker J, et al: Comprehensive assessment of
                    153. Pagliuca FW, Millman JR, Gurtler M, et al: Generation of functional human pancreatic   HIV target cells in the distal human gut suggests increasing HIV susceptibility toward
                     beta cells in vitro. Cell 159(2):428–439, 2014.       the anus. J Acquir Immune Defic Syndr 63(3):263–271, 2013.
                    154. Collombat P, Xu X, Ravassard P, et al: The ectopic expression of Pax4 in the mouse     172. Holt N, Wang J, Kim K, et al: Human hematopoietic stem/progenitor cells modi-
                     pancreas converts progenitor cells into alpha and subsequently beta cells. Cell 138(3):   fied by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol
                     449–462, 2009.                                        28(8):839–847, 2010.
                    155. Thorel F, Nepote V, Avril I, et al: Conversion of adult pancreatic alpha-cells to beta-cells     173. Hofer U, Henley JE, Exline CM, et al: Pre-clinical modeling of CCR5 knockout in
                     after extreme beta-cell loss. Nature 464(7292):1149–1154, 2010.  human hematopoietic stem cells by zinc finger nucleases using humanized mice. J Infect
                    156. Hacein-Bey-Abina S, Garrigue A, Wang GP, et al: Insertional oncogenesis in 4 patients   Dis 208 Suppl 2:S160–S164, 2013.
                     after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118(9):3132–3142,     174. Allers K, Hutter G, Hofmann J, et al: Evidence for the cure of HIV infection by
                     2008.                                                 CCR5Delta32/Delta32 stem cell transplantation. Blood 117(10):2791–2799, 2011.
                    157. Cartier  N,  Hacein-Bey-Abina  S, Bartholomae  CC,  et  al:  Hematopoietic  stem cell     175. Hutter G, Nowak D, Mossner M, et al: Long-term control of HIV by CCR5 Delta32/
                     gene  therapy  with  a  lentiviral  vector  in  X-linked  adrenoleukodystrophy.  Science   Delta32 stem-cell transplantation. N Engl J Med 360(7):692–698, 2009.
                     326(5954):818–823, 2009.                             176. de Mendoza C, Rodriguez C, Garcia F, et al: Prevalence of X4 tropic viruses in patients
                    158. Biffi A, Montini E, Lorioli L, et al: Lentiviral hematopoietic stem cell gene therapy ben-  recently infected with HIV-1 and lack of association with transmission of drug resis-
                     efits metachromatic leukodystrophy. Science 341(6148):1233158, 2013.  tance. J Antimicrob Chemother 59(4):698–704, 2007.
                    159. Aiuti A, Biasco L, Scaramuzza S, et al: Lentiviral hematopoietic stem cell gene therapy     177. Didigu CA, Wilen CB, Wang J, et al: Simultaneous zinc-finger nuclease editing of the
                     in patients with Wiskott-Aldrich syndrome. Science 341(6148):1233151, 2013.  HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection. Blood
                    160. Lombardo A, Genovese P, Beausejour CM, et al: Gene editing in human stem cells using   123(1):61–69, 2014.
                     zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol     178. Provasi E, Genovese P, Lombardo A, et al: Editing T cell specificity towards leukemia by
                     25(11):1298–1306, 2007.                               zinc finger nucleases and lentiviral gene transfer. Nat Med 18(5):807–815, 2012.
                    161. Hockemeyer D, Soldner F, Beard C, et al: Efficient targeting of expressed and silent     179. Porter DL, Levine BL, Kalos M, et al: Chimeric antigen receptor-modified T cells in
                     genes in human ESCs and iPSCs using zinc-finger nucleases.  Nat Biotechnol 27(9):   chronic lymphoid leukemia. N Engl J Med 365(8):725–733, 2011.
                     851–857, 2009.                                       180. Torikai H, Reik A, Liu PQ, et al: A foundation for universal T-cell based immuno-
                    162. DeKelver RC, Choi VM, Moehle EA, et al: Functional genomics, proteomics, and regu-  therapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and
                     latory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis   eliminate expression of endogenous TCR. Blood 119(24):5697–5705, 2012.
                     into a safe harbor locus in the human genome. Genome Res 20(8):1133–1142, 2010.    181. Liang G, Zhang Y: Genetic and epigenetic variations in iPSCs: Potential causes and
                    163. Lombardo A, Cesana D, Genovese P, et al: Site-specific integration and tailoring of cas-  implications for application. Cell Stem Cell 13(2):149–159, 2013.
                     sette design for sustainable gene transfer. Nat Methods 8(10):861–869, 2011.    182. Amps K, Andrews PW, Anyfantis G, et al: Screening ethnically diverse human embry-
                    164. Soldner F, Laganiere J, Cheng AW, et al: Generation of isogenic pluripotent stem cells   onic stem cells identifies a chromosome 20 minimal amplicon conferring growth
                     differing exclusively at two early onset Parkinson point mutations. Cell 146(2):318–331,   advantage. Nat Biotechnol 29(12):1132–1144, 2011.
                     2011.                                                183. Ben-David U, Benvenisty N: High prevalence of evolutionarily conserved and
                    165. Zou J, Mali P, Huang X, Dowey SN, Cheng L: Site-specific gene correction of a point   species-specific genomic aberrations in mouse pluripotent stem cells.  Stem Cells
                     mutation in human iPS cells derived from an adult patient with sickle cell disease. Blood   30(4):612–622, 2012.
                     118(17):4599–4608, 2011.                             184. Smith C, Gore A, Yan W, et al: Whole-genome sequencing analysis reveals high spec-
                    166. Genovese P, Schiroli G, Escobar G, et al: Targeted genome editing in human repopulat-  ificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs. Cell Stem
                     ing haematopoietic stem cells. Nature 510(7504):235–240, 2014.  Cell 15(1):12–13, 2014.
                    167. Boitano AE, Wang J, Romeo R, et al: Aryl hydrocarbon receptor antagonists promote     185. Osborn MJ, Starker CG, McElroy AN, et al: TALEN-based gene correction for epider-
                     the expansion of human hematopoietic stem cells. Science 329(5997):1345–1348, 2010.  molysis bullosa. Mol Ther 21(6):1151–1159, 2013.
                    168. North TE, Goessling W, Walkley CR, et al: Prostaglandin E2 regulates vertebrate hae-
                     matopoietic stem cell homeostasis. Nature 447(7147):1007–1011, 2007.











































          Kaushansky_chapter 30_p0447-0458.indd   457                                                                   9/17/15   6:08 PM
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