دورية أكاديمية
أعرض في EDS

Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin.

التفاصيل البيبلوغرافية
العنوان: Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin.
المؤلفون: Palchaudhuri R; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, Massachusetts, USA., Saez B; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA., Hoggatt J; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA., Schajnovitz A; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA., Sykes DB; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA., Tate TA; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA., Czechowicz A; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA.; Program in Cellular and Molecular Medicine, Division of Hematology/Oncology, Boston Children's Hospital, Boston, Massachusetts, USA.; Department of Pediatrics, Harvard Medical School, Boston, Massachusetts, USA.; Department of Pediatric Oncology, Dana Farber Cancer Institute, Boston, Massachusetts, USA., Kfoury Y; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA., Ruchika F; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA., Rossi DJ; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA.; Program in Cellular and Molecular Medicine, Division of Hematology/Oncology, Boston Children's Hospital, Boston, Massachusetts, USA.; Department of Pediatrics, Harvard Medical School, Boston, Massachusetts, USA., Verdine GL; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, Massachusetts, USA., Mansour MK; Division of Infectious Diseases, Massachusetts General Hospital, Boston, Massachusetts, USA., Scadden DT; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts, USA.; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Harvard Stem Cell Institute, Cambridge, Massachusetts, USA.
المصدر: Nature biotechnology [Nat Biotechnol] 2016 Jul; Vol. 34 (7), pp. 738-45. Date of Electronic Publication: 2016 Jun 06.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Nature America Publishing Country of Publication: United States NLM ID: 9604648 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-1696 (Electronic) Linking ISSN: 10870156 NLM ISO Abbreviation: Nat Biotechnol Subsets: MEDLINE
أسماء مطبوعة: Publication: New York Ny : Nature America Publishing
Original Publication: New York, NY : Nature Pub. Co., [1996-
مواضيع طبية MeSH: DNA Damage/*immunology , Hematopoietic Stem Cell Transplantation/*methods , Hematopoietic Stem Cells/*immunology , Leukocyte Common Antigens/*immunology , Ribosome Inactivating Proteins, Type 1/*genetics , Ribosome Inactivating Proteins, Type 1/*immunology, Animals ; Antibodies, Monoclonal/genetics ; Antibodies, Monoclonal/immunology ; Cell Differentiation/genetics ; Cell Differentiation/immunology ; Cell Survival/genetics ; Cell Survival/immunology ; Cells, Cultured ; DNA Damage/genetics ; Female ; Genetic Enhancement/methods ; Immunogenetic Phenomena/genetics ; Immunotoxins ; Mice ; Mice, Inbred C57BL ; Saporins
مستخلص: Hematopoietic stem cell transplantation (HSCT) offers curative therapy for patients with hemoglobinopathies, congenital immunodeficiencies, and other conditions, possibly including AIDS. Autologous HSCT using genetically corrected cells would avoid the risk of graft-versus-host disease (GVHD), but the genotoxicity of conditioning remains a substantial barrier to the development of this approach. Here we report an internalizing immunotoxin targeting the hematopoietic-cell-restricted CD45 receptor that effectively conditions immunocompetent mice. A single dose of the immunotoxin, CD45-saporin (SAP), enabled efficient (>90%) engraftment of donor cells and full correction of a sickle-cell anemia model. In contrast to irradiation, CD45-SAP completely avoided neutropenia and anemia, spared bone marrow and thymic niches, enabling rapid recovery of T and B cells, preserved anti-fungal immunity, and had minimal overall toxicity. This non-genotoxic conditioning method may provide an attractive alternative to current conditioning regimens for HSCT in the treatment of non-malignant blood diseases.
Competing Interests: The authors declare competing financial interests: details are available in the online version of the paper.
التعليقات: Comment in: Nat Biotechnol. 2016 Jul 12;34(7):721-3. doi: 10.1038/nbt.3629. (PMID: 27404882)
Comment in: Mol Ther. 2016 Nov;24(11):1892-1894. doi: 10.1038/mt.2016.193. (PMID: 27916993)
References: Br J Haematol. 1996 Jun;93(4):789-94. (PMID: 8703804)
Infection. 2012 Jun;40(3):271-8. (PMID: 22187340)
Blood. 2003 Mar 15;101(6):2434-9. (PMID: 12433683)
Science. 2007 Nov 23;318(5854):1296-9. (PMID: 18033883)
Diabetes. 2014 Sep;63(9):3041-6. (PMID: 24947362)
Cancer Immunol Immunother. 2008 Dec;57(12):1879-90. (PMID: 18408925)
Sci Transl Med. 2012 Mar 7;4(124):124ra28. (PMID: 22399264)
J Infect Dis. 1999 Mar;179 Suppl 2:S318-25. (PMID: 10081502)
Lancet. 2009 Sep 12;374(9693):912-20. (PMID: 19729196)
Curr Opin Hematol. 2013 Nov;20(6):501-8. (PMID: 24104410)
Blood. 1996 Apr 15;87(8):3494-9. (PMID: 8605369)
J Exp Med. 1983 Apr 1;157(4):1077-88. (PMID: 6220106)
J Immunol Methods. 2007 Dec 1;328(1-2):204-14. (PMID: 17804011)
Development. 2014 Dec;141(24):4656-66. (PMID: 25468935)
Blood. 2001 Dec 1;98(12):3456-64. (PMID: 11719388)
Bone Marrow Transplant. 2000 Feb;25(4):401-4. (PMID: 10723583)
Int Immunol. 2000 Mar;12(3):313-24. (PMID: 10700466)
Jpn J Infect Dis. 2004 Oct;57(5):S27-8. (PMID: 15507764)
Blood. 2005 Dec 1;106(12):3854-9. (PMID: 16105980)
Hematology Am Soc Hematol Educ Program. 2009;:690-7. (PMID: 20008255)
Blood. 2009 Sep 10;114(11):2333-43. (PMID: 19433859)
Nat Rev Cancer. 2006 Jul;6(7):559-65. (PMID: 16794638)
Front Pharmacol. 2015 Jan 12;5:278. (PMID: 25628564)
Blood. 2006 Mar 1;107(5):2184-91. (PMID: 16254140)
J Pediatr Hematol Oncol. 2015 Jul;37(5):e295-300. (PMID: 25985240)
Blood. 2010 Dec 9;116(24):5419-22. (PMID: 20813896)
Proc Natl Acad Sci U S A. 2007 Jan 2;104(1):175-80. (PMID: 17190805)
Blood. 2001 Jun 15;97(12):3960-5. (PMID: 11389040)
N Engl J Med. 2009 Feb 12;360(7):692-8. (PMID: 19213682)
N Engl J Med. 2009 Dec 10;361(24):2309-17. (PMID: 20007560)
Methods Mol Biol. 2013;979:147-59. (PMID: 23397394)
Ann N Y Acad Sci. 2003 May;996:80-8. (PMID: 12799286)
Science. 1990 Feb 2;247(4942):566-8. (PMID: 2154033)
Bone Marrow Transplant. 2005 Dec;36(11):971-5. (PMID: 16205730)
Curr Opin Mol Ther. 2008 Oct;10(5):471-8. (PMID: 18830923)
Prostate. 1998 Mar 1;34(4):259-69. (PMID: 9496900)
Blood. 2010 May 20;115(20):4120-9. (PMID: 20231424)
Trends Mol Med. 2016 Apr;22(4):317-27. (PMID: 26993219)
Clin Transl Sci. 2009 Aug;2(4):279-85. (PMID: 19750208)
Biol Blood Marrow Transplant. 2001;7(12):665-73. (PMID: 11787529)
Blood. 2006 May 1;107(9):3764-71. (PMID: 16439683)
J Immunol. 2009 Aug 15;183(4):2506-12. (PMID: 19625646)
J Immunol Methods. 2002 Aug 1;266(1-2):19-32. (PMID: 12133619)
Hematology Am Soc Hematol Educ Program. 2006;:398-401. (PMID: 17124089)
JAMA. 2014 Jun 25;311(24):2490-8. (PMID: 25058083)
Science. 2013 Aug 23;341(6148):1233158. (PMID: 23845948)
Blood. 2012 Feb 2;119(5):1130-8. (PMID: 22134165)
Blood. 1994 Mar 1;83(5):1390-7. (PMID: 8118040)
Blood. 2008 Oct 1;112(7):2858-68. (PMID: 18544681)
Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S51-3. (PMID: 18978745)
Hum Gene Ther. 2014 Dec;25(12):1013-22. (PMID: 24937231)
Leukemia. 2004 Mar;18(3):575-83. (PMID: 14749701)
Blood. 2014 Nov 6;124(19):2937-47. (PMID: 25202142)
Appl Immunohistochem Mol Morphol. 2005 Sep;13(3):205-20. (PMID: 16082245)
Science. 2013 Aug 23;341(6148):1233151. (PMID: 23845947)
J Leukoc Biol. 1999 Dec;66(6):996-1004. (PMID: 10614783)
Nature. 2009 Jan 1;457(7225):92-6. (PMID: 19052546)
Clin Cancer Res. 2014 Feb 1;20(3):617-30. (PMID: 24097861)
Bioorg Med Chem Lett. 2014 Dec 1;24(23):5357-63. (PMID: 25455482)
Hematology Am Soc Hematol Educ Program. 2012;2012:265-70. (PMID: 23233590)
Blood. 2015 Jun 11;125(24):3798-804. (PMID: 25824692)
Leuk Lymphoma. 2004 Feb;45(2):229-36. (PMID: 15101706)
Cancer Genomics Proteomics. 2014 Jan-Feb;11(1):25-38. (PMID: 24633317)
معلومات مُعتمدة: T32 HL007574 United States HL NHLBI NIH HHS; R01 HL044851 United States HL NHLBI NIH HHS; R01 HL107630 United States HL NHLBI NIH HHS; K08 AI110655 United States AI NIAID NIH HHS; K99 HL119559 United States HL NHLBI NIH HHS; R00 HL119559 United States HL NHLBI NIH HHS; U19 HL129903 United States HL NHLBI NIH HHS
المشرفين على المادة: 0 (Antibodies, Monoclonal)
0 (Immunotoxins)
0 (Ribosome Inactivating Proteins, Type 1)
EC 3.1.3.48 (Leukocyte Common Antigens)
EC 3.1.3.48 (Ptprc protein, mouse)
EC 3.2.2.22 (Saporins)
تواريخ الأحداث: Date Created: 20160609 Date Completed: 20170605 Latest Revision: 20240610
رمز التحديث: 20240610
مُعرف محوري في PubMed: PMC5179034
DOI: 10.1038/nbt.3584
PMID: 27272386
قاعدة البيانات: MEDLINE
كن أول من يترك تعليقا!
يجب تسجيل دخولك أولا