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1558   Part IX  Cell-Based Therapies


        an appropriate donor, eliminates the risk of GVHD, and in some   Laufs  S,  Nagy  KZ,  Giordano  FA,  et al:  Insertion  of  retroviral  vectors  in
        cases  reduces  the  intensity  of  preparative  regimen  required  before   NOD/SCID  repopulating  human  peripheral  blood  progenitor  cells
        transplant, which also reduces toxicity. Thus, in some diseases, this   occurs preferentially in the vicinity of transcription start regions and in
        therapeutic approach can now be considered an alternative to stan-  introns. Mol Ther 10:874–881, 2004.
        dard therapy. Insertional mutagenesis, which has resulted in serious   Li  Z,  Dullmann  J,  Schiedlmeier  B,  et al:  Murine  leukemia  induced  by
        adverse events in several trials, has stimulated rapid development of   retroviral gene marking. Science 296:497, 2002.
        putative safer vector systems that are being tested in human trials but   Milsom  M,  Schambach  A,  Williams  DA,  et al:  Chemoprotective  gene
        remains a challenge. Rapid progress in molecular technology, such as   delivery. In Harington KJ, Vile RG, Pandha H, editors: Viral therapy of
        high-throughput sequencing and the development of new sources of   cancer, Indianapolis, IN, 2008, Wiley Press, pp 376–391.
        expandable stem cell sources, offers significant potential for ongoing   Miyoshi H, Smith KA, Mosier DE, et al: Transduction of human CD34(+)
        development of gene transfer in regenerative biology for a wide range   cells that mediate long-term engraftment of NOD/SCID mice by HIV
        of human conditions.                                     vectors. Science 283:682–686, 1999.
                                                              Moritz T, Patel VP, Williams DA: Bone marrow extracellular matrix molecules
                                                                 improve  gene  transfer  into  human  hematopoietic  cells  via  retroviral
        ACKNOWLEDGEMENTS                                         vectors. J Clin Invest 93:1451–1457, 1994.
                                                              Naldini L, Blomer U, Gallay P, et al: In vivo gene delivery and stable trans-
        Funding from NIH DK062757, CA113969, AI097628, DK090913,   duction of nondividing cells by a lentiviral vector. Science 272:263–267,
        and the NHLBI Gene Therapy Resource Program. I would like to   1996.
        thank members of my laboratory and members of the Transatlantic   Ott MG, Stein S, Schultze-Strasser S, et al: Phase I/II gene therapy study for
        Gene Therapy Consortium for productive collaborations.   Chronic Granulomatous Disease: results, lessons and perspectives. Blood
                                                                 (ASH Annual Meeting Abstracts) 110:503, 2007.
                                                              Pai  S,  Notarangelo  L,  Harris  C,  et al:  Somatic  gene  therapy  for  X-linked
        SUGGESTED READINGS                                       severe  combined  immunodeficiency  using  a  self-inactivating  modified
                                                                 gammaretroviral vector results in an improved preclinical safety profile
        Aiuti A, Cassani B, Andolfi G, et al: Multilineage hematopoietic reconstitu-  and early clinical efficacy in a human patient. Blood (ASH Annual Meeting
           tion without clonal selection in ADA-SCID patients treated with stem   Abstracts) 118:164, 2011.
           cell gene therapy. J Clin Invest 117:2233–2240, 2007.  Park IH, Zhao R, West JA, et al: Reprogramming of human somatic cells to
        Aiuti  A,  Slavin  S,  Aker  M,  et al:  Correction  of  ADA-SCID  by  stem  cell   pluripotency with defined factors. Nature 451:141–146, 2008.
           gene  therapy  combined  with  nonmyeloablative  conditioning.  Science   Porteus  MH,  Carroll  D:  Gene  targeting  using  zinc  finger  nucleases.  Nat
           296:2410–2413, 2002.                                  Biotechnol 23:967–973, 2005.
        Bauer TR, Jr, Allen JM, Hai M, et al: Successful treatment of canine leukocyte   Sadelain M, Papapetrou EP, Bushman FD: Safe harbours for the integration
           adhesion deficiency by foamy virus vectors. Nat Med 14:93–97, 2008.  of new DNA in the human genome. Nat Rev Cancer 12:51–58, 2012.
        Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al: Hematopoietic stem   Sankaran VG, Menne TF, Xu J, et al: Human fetal hemoglobin expression is
           cell gene therapy with a lentiviral vector in X-linked adrenoleukodystro-  regulated by the developmental stage-specific repressor BCL11A. Science
           phy. Science 326:818–823, 2009.                       322:1839–1842, 2008.
        Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al: Gene therapy of   Stein S, Ott MG, Schultze-Strasser S, et al: Genomic instability and myelo-
           human severe combined immunodeficiency (SCID)-X1 disease. Science   dysplasia  with  monosomy  7  consequent  to  EVI1  activation  after  gene
           288:669–672, 2000.                                    therapy for chronic granulomatous disease. Nat Med 16:198–204, 2010.
        Cavazzana-Calvo  M,  Payen  E,  Negre  O,  et al:  Transfusion  independence   Takahashi K, Tanabe K, Ohnuki M, et al: Induction of pluripotent stem cells
           and HMGA2 activation after gene therapy of human beta-thalassaemia.   from adult human fibroblasts by defined factors. Cell 131:861–872, 2007.
           Nature 467:318–322, 2010.                          Thrasher AJ, Gaspar HB, Baum C, et al: Gene therapy: X-SCID transgene
        Gaspar  HB,  Parsley  KL,  Howe  S,  et al:  Gene  therapy  of  X-linked  severe   leukaemogenicity. Nature 443:E5–E6, discussion E6-E7, 2006.
           combined immunodeficiency by use of a pseudotyped gammaretroviral   Urnov FD, Miller JC, Lee YL, et al: Highly efficient endogenous human gene
           vector. Lancet 364:2181–2187, 2004.                   correction  using  designed  zinc-finger  nucleases.  Nature  435:646–651,
        Hacein-Bey-Abina  S,  Le  Deist  F,  Carlier  F,  et al:  Sustained  correction  of   2005.
           X-linked severe combined immunodeficiency by ex vivo gene therapy. N   Williams  DA,  Thrasher  AJ:  Concise  review:  lessons  learned  from  clinical
           Engl J Med 346:1185–1193, 2002.                       trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells
        Hacein-Bey-Abina  S,  Von  Kalle  C,  Schmidt  M,  et al:  LMO2-associated   Transl Med 3:636–642, 2014.
           clonal T cell proliferation in two patients after gene therapy for SCID-X1.   Xu  J,  Peng  C,  Sankaran  VG,  et al:  Correction  of  sickle  cell  disease  in
           Science 302:415–419, 2003.                            adult  mice  by  interference  with  fetal  hemoglobin  silencing.  Science
        Hanna J, Wernig M, Markoulaki S, et al: Treatment of sickle cell anemia   334:993–996, 2011.
           mouse  model  with  iPS  cells  generated  from  autologous  skin.  Science   Yu  J,  Vodyanik  MA,  Smuga-Otto  K,  et al:  Induced  pluripotent  stem  cell
           318:1920–1923, 2007.                                  lines derived from human somatic cells. Science 318:1917–1920, 2007.
        Hirschhorn R, Yang DR, Puck JM, et al: Spontaneous in vivo reversion to
           normal of an inherited mutation in a patient with adenosine deaminase
           deficiency. Nat Genet 13:290–295, 1996.            REFERENCES
        Howe SJ, Mansour MR, Schwarzwaelder K, et al: Insertional mutagenesis
           combined with acquired somatic mutations causes leukemogenesis fol-  For the complete list of references, log on to www.expertconsult.com.
           lowing gene therapy of SCID-X1 patients. J Clin Invest 118:3143–3150,
           2008.
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