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Stem cell transplantation

A non-myeloablative conditioning approach for long-term engraftment of human and mouse hematopoietic stem cells

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  1. 1.

    Stanevsky A, Goldstein G, Nagler A. Umbilical cord blood transplantation: pros, cons and beyond. Blood. 2009;23:199–204.

  2. 2.

    Pusic I, Jiang SY, Landua S, Uy GL, Rettig MP, Cashen AF, et al. Impact of mobilization and remobilization strategies on achieving sufficient stem cell yields for autologous transplantation. Biol Blood Marrow Transplant. 2008;14:1045–56.

  3. 3.

    Stewart FM, Zhong S, Wuu J, Hsieh C, Nilsson SK, Quesenberry PJ. Lymphohematopoietic engraftment in minimally myeloablated hosts. Blood. 1998;91:3681–7.

  4. 4.

    Czechowicz A, Kraft D, Weissman IL, Bhattacharya D. Efficient transplantation via antibody-based clearance of hematopoietic stem cell niches. Science. 2007;318:1296–9.

  5. 5.

    Quesenberry PJ, Stewart FM, Becker P, D’Hondt L, Frimberger A, Lambert JF, et al. Stem cell engraftment strategies. Ann N Y Acad Sci. 2001;938:54–61.

  6. 6.

    Xue X, Pech NK, Shelley WC, Srour EF, Yoder MC, Dinauer MC. Antibody targeting KIT as pretransplantation conditioning in immunocompetent mice. Blood. 2010;116:5419–22.

  7. 7.

    Chandrakasan S, Jayavaradhan R, Ernst J, Shrestha A, Loberg A, Dexheimer P, et al. KIT blockade is sufficient for donor hematopoietic stem cell engraftment in Fanconi anemia mice. Blood. 2017;129:1048–52.

  8. 8.

    Wang Z, Li G, Tse W, Bunting KD. Conditional deletion of STAT5 in adult mouse hematopoietic stem cells causes loss of quiescence and permits efficient nonablative stem cell replacement. Blood. 2009;113:4856–65.

  9. 9.

    Cao LQ, Liu L, Xu LP, Zhang XH, Wang Y, Fan QZ, et al. Correlation between pediatric donor characteristics and cell compositions in mixture allografts of combined G-CSF-mobilized PBSCs and bone marrow allografts. Bone Marrow Transplant. 2018;53:108–10.

  10. 10.

    Dar A, Schajnovitz A, Lapid K, Kalinkovich A, Itkin T, Ludin A, et al. Rapid mobilization of hematopoietic progenitors by AMD3100 and catecholamines is mediated by CXCR4-dependent SDF-1 release from bone marrow stromal cells. Leukemia. 2011;25:1286–96.

  11. 11.

    Yang L, Wang L, Geiger H, Cancelas JA, Mo J, Zheng Y. Rho GTPase Cdc42 coordinates hematopoietic stem cell quiescence and niche interaction in the bone marrow. Proc Natl Acad Sci USA. 2007;104:5091–6.

  12. 12.

    Liu W, Du W, Shang X, Wang L, Evelyn C, Florian MC et al. Rational identification of a Cdc42 inhibitor presents a new regimen for long-term hematopoietic stem cell mobilization. Leukemia. Under review.

  13. 13.

    Thakar MS, Kurre P, Storb R, Kletzel M, Frangoul H, Pulsipher MA, et al. Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation. Bone Marrow Transplant. 2011;46:539–44.

  14. 14.

    Notta F, Doulatov S, Laurenti E, Poeppl A, Jurisica I, Dick JE. Isolation of single human hematopoietic stem cells capable of long-term multilineage engraftment. Science. 2011;333:218–21.

  15. 15.

    Adair JE, Becker PS, Chandrasekaran D, Choi G, Woolfrey AE, Burroughs L, et al. Gene therapy for fanconi anemia in Seattle: clinical experience and next steps. Blood. 2016;128:3510.

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We thank Dr. Madeleine Carreau (Laval University) for the Fanca+/ mice, the Vector Core of the Cincinnati Children’s Research Foundation (Cincinnati Children’s Hospital Medical Center) for the preparation of retroviruses, and the Comprehensive Mouse and Cancer Core of the Cincinnati Children’s Research Foundation (Cincinnati Children’s Hospital Medical Center) for bone marrow transplantation service. This work was supported by NIH grants R01 CA193350, R01 DK104814, R01 CA150547, R01 HL076712, and R01 HD089932. Q.P. and J.M. were Leukemia and Lymphoma Scholar. W.D. was supported by the NIH T32 HL091805 training grant. The studies were further supported by a NIH center grant P30 DK090971.

Author information

W.D. and W.L. designed and performed research, analyzed data, and wrote the paper; X.S., B.M., and J.S. performed research; M.W. and J.M. contributed vital new reagents or analytical tools; S.D. and H.G. wrote the paper; Q.P. and Y.Z. designed research, contributed vital new reagents or analytical tools, analyzed data, and wrote the paper.

Conflict of interest

The authors declare that they have no conflict of interest.

Correspondence to Wei Du or Qishen Pang or Yi Zheng.

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