Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

被引：34

作者：

Cavazzana-Calvo, Marina ^{[1
,2
,3
]}

Fischer, Alain ^{[2
,3
,4
]}

Bushman, Frederic D. ^{[5
]}

Payen, Emmanuel ^{[6
,7
,8
]}

Hacein-Bey-Abina, Salima ^{[1
,2
,3
]}

Leboulch, Philippe ^{[6
,7
,8
,9
,10
]}

机构：

[1] Hop Necker Enfants Malad, AP HP, Dept Biotherapy, F-75015 Paris, France

[2] Hop Necker Enfants Malad, Inserm U 768, F-75015 Paris, France

[3] Univ Paris 05, Fac Med, IFR94, Paris, France

[4] Hop Necker Enfants Malad, AP HP, Dept Pediat Immunohaematol, F-75015 Paris, France

[5] Univ Penn, Sch Med, Dept Microbiol, Philadelphia, PA 19104 USA

[6] CEA, Inst Emerging Dis & Innovat Therapies IMETI, Fontenay Aux Roses, France

[7] Univ Paris 11, Commissariat Energie Atom & Energies Alternat CEA, Inst Therapies Innovants iMETI, Fontenay Aux Roses, France

[8] INSERM, U962, Fontenay Aux Roses, France

[9] Brigham & Womens Hosp, Div Genet, Boston, MA 02115 USA

[10] Harvard Univ, Sch Med, Boston, MA USA

来源：

BLOOD | 2011年 / 117卷 / 17期

基金：

美国国家卫生研究院;

关键词：

SEVERE COMBINED IMMUNODEFICIENCY; GENE-THERAPY; SELF-RENEWAL; IN-VIVO; LINEAGE COMMITMENT; DIFFERENTIATION; COMPARTMENT; TRANSPLANTATION; RECONSTITUTION; REPOPULATION;

D O I：

10.1182/blood-2010-09-255679

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

The understanding of the hierarchical organization of the human hematopoietic system is of major biologic and clinical significance. The validity of the conventional model in which hematopoiesis is solely maintained by a pool of multipotent long-term hematopoietic stem cells (LT-HSCs) has been recently challenged by several mouse studies. These new data point to the existence of a heterogeneous stem cell population that consists of distinct subsets of LT-HSCs, which include stem cells biased toward lineage-specific differentiation programs. This review attempts to discuss the balanced versus biased patterns of lineage output of human LT-HSCs gathered in 3 different gene therapy trials on the basis of vector integration site analysis by deep sequencing. The distribution of integration sites observed tends to support the validity of the revised model. (Blood. 2011; 117(17): 4420-4424)

引用

页码：4420 / 4424

页数：5

共 36 条

[1] Identification of Flt3+ lympho-myeloid stem cells lacking erythro-megakaryocytic potential:: A revised road map for adult blood lineage commitment [J].

Adolfsson, J ;

Månsson, R ;

Buza-Vidas, N ;

Hultquist, A ;

Liuba, K ;

Jensen, CT ;

Bryder, D ;

Yang, LP ;

Borge, OJ ;

Thoren, LAM ;

Anderson, K ;

Sitnicka, E ;

Sasaki, Y ;

Sigvardsson, M ;

Jacobsen, SEW .

CELL, 2005, 121 (02) :295-306

[2] Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. [J].

Aiuti, Alessandro ;

Cattaneo, Federica ;

Galimberti, Stefania ;

Benninghoff, Ulrike ;

Cassani, Barbara ;

Callegaro, Luciano ;

Scaramuzza, Samantha ;

Andolfi, Grazia ;

Mirolo, Massimiliano ;

Brigida, Immacolata ;

Tabucchi, Antonella ;

Carlucci, Filippo ;

Eibl, Martha ;

Aker, Memet ;

Slavin, Shimon ;

Al-Mousa, Hamoud ;

Al Ghonaium, Abdulaziz ;

Ferster, Alina ;

Duppenthaler, Andrea ;

Notarangelo, Luigi ;

Wintergerst, Uwe ;

Buckley, Rebecca H. ;

Bregni, Marco ;

Marktel, Sarah ;

Valsecchi, Maria Grazia ;

Rossi, Paolo ;

Ciceri, Fabio ;

Miniero, Roberto ;

Bordignon, Claudio ;

Roncarolo, Maria-Grazia .

NEW ENGLAND JOURNAL OF MEDICINE, 2009, 360 (05) :447-458

[3] Reciprocal activation of GATA-1 and PU.1 marks initial specification of hematopoietic stem cells into myeloerythroid and myelolymphoid lineages [J].

Arinobu, Yojiro ;

Mizuno, Shin-ichi ;

Chong, Yong ;

Shigematsu, Hirokazu ;

Lino, Tadafumi ;

Iwasaki, Hiromi ;

Graf, Thomas ;

Mayfield, Robin ;

Chan, Susan ;

Kastner, Philippe ;

Akashi, Koichi .

CELL STEM CELL, 2007, 1 (04) :416-427

[4] Hematopoietic stem cells - The paradigmatic tissue-specific stem cell [J].

Bryder, David ;

Rossi, Derrick J. ;

Weissman, Irving L. .

AMERICAN JOURNAL OF PATHOLOGY, 2006, 169 (02) :338-346

[5] Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy [J].

Cartier, Nathalie ;

Hacein-Bey-Abina, Salima ;

Bartholomae, Cynthia C. ;

Veres, Gabor ;

Schmidt, Manfred ;

Kutschera, Ina ;

Vidaud, Michel ;

Abel, Ulrich ;

Dal-Cortivo, Liliane ;

Caccavelli, Laure ;

Mahlaoui, Nizar ;

Kiermer, Veronique ;

Mittelstaedt, Denice ;

Bellesme, Celine ;

Lahlou, Najiba ;

Lefrere, Francois ;

Blanche, Stephane ;

Audit, Muriel ;

Payen, Emmanuel ;

Leboulch, Philippe ;

l'Homme, Bruno ;

Bougneres, Pierre ;

Von Kalle, Christof ;

Fischer, Alain ;

Cavazzana-Calvo, Marina ;

Aubourg, Patrick .

SCIENCE, 2009, 326 (5954) :818-823

[6] Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype [J].

Cavazzana-Calvo, Marina ;

Carlier, Frederique ;

Le Deist, Francoise ;

Morillon, Estelle ;

Taupin, Pierre ;

Gautier, David ;

Radford-Weiss, Isabelle ;

Caillat-Zucman, Sophie ;

Neven, Benedicte ;

Blanche, Stephane ;

Cheynier, Remi ;

Fischer, Alain ;

Hacein-Bey-Abina, Salima .

BLOOD, 2007, 109 (10) :4575-4581

[7] Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia [J].

Cavazzana-Calvo, Marina ;

Payen, Emmanuel ;

Negre, Olivier ;

Wang, Gary ;

Hehir, Kathleen ;

Fusil, Floriane ;

Down, Julian ;

Denaro, Maria ;

Brady, Troy ;

Westerman, Karen ;

Cavallesco, Resy ;

Gillet-Legrand, Beatrix ;

Caccavelli, Laure ;

Sgarra, Riccardo ;

Maouche-Chretien, Leila ;

Bernaudin, Francoise ;

Girot, Robert ;

Dorazio, Ronald ;

Mulder, Geert-Jan ;

Polack, Axel ;

Bank, Arthur ;

Soulier, Jean ;

Larghero, Jerome ;

Kabbara, Nabil ;

Dalle, Bruno ;

Gourmel, Bernard ;

Socie, Gerard ;

Chretien, Stany ;

Cartier, Nathalie ;

Aubourg, Patrick ;

Fischer, Alain ;

Cornetta, Kenneth ;

Galacteros, Frederic ;

Beuzard, Yves ;

Gluckman, Eliane ;

Bushman, Frederick ;

Hacein-Bey-Abina, Salima ;

Leboulch, Philippe .

NATURE, 2010, 467 (7313) :318-U94

[8] Modulation of p210BCR-ABL activity in transduced primary human hematopoietic cells controls lineage programming [J].

Chalandon, Y ;

Jiang, XY ;

Hazlewood, G ;

Loutet, S ;

Conneally, E ;

Eaves, A ;

Eaves, C .

BLOOD, 2002, 99 (09) :3197-3204

[9] Distinct Hematopoietic Stem Cell Subtypes Are Differentially Regulated by TGF-β1 [J].

Challen, Grant A. ;

Boles, Nathan C. ;

Chambers, Stuart M. ;

Goodell, Margaret A. .

CELL STEM CELL, 2010, 6 (03) :265-278

[10] A new mechanism for the aging of hematopoietic stem cells: aging changes the clonal composition of the stem cell compartment but not individual stem cells [J].

Cho, Rebecca H. ;

Sieburg, Hans B. ;

Muller-Sieburg, Christa E. .

BLOOD, 2008, 111 (12) :5553-5561

← 1 2 3 4 →