Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia

被引：21

作者：

Mueller, Lars U. W. ^{[1
,2
,3
]}

Williams, David A. ^{[1
,2
,3
]}

机构：

[1] Harvard Univ, Sch Med, Childrens Hosp Boston, Dept Med,Div Pediat Hematol Oncol, Boston, MA 02115 USA

[2] Harvard Univ, Sch Med, Dana Farber Canc Inst, Boston, MA 02115 USA

[3] Harvard Univ, Sch Med, Harvard Stem Cell Inst, Boston, MA 02115 USA

来源：

MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS | 2009年 / 668卷 / 1-2期

关键词：

Fanconi anemia; HSC; Gene therapy; Bone marrow failure; COLONY-STIMULATING FACTOR; DOSE G-CSF; BLOOD CD34(+) CELLS; SEVERE COMBINED IMMUNODEFICIENCY; BONE-MARROW-TRANSPLANTATION; NATURAL GENE-THERAPY; PLUS G-CSF; PROGENITOR CELLS; ENHANCES ENGRAFTMENT; TARGETED DISRUPTION;

D O I：

10.1016/j.mrfmmm.2009.03.010

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Fanconi anemia is a rare bone marrow failure and cancer predisposition syndrome. Childhood onset of aplastic anemia is one of the hallmarks of this condition. Supportive therapy in the form of blood products, androgens, and hematopoietic growth factors may boost blood counts temporarily. However, allogeneic hematopoietic stem cell transplantation (HSCT) currently remains the only curative treatment option for the hematologic manifestations of Fanconi anemia (FA). Here we review current clinical and pre-clinical strategies for treating hematopoietic stem cell (HSC) failure, including the experience with mobilizing and collecting CD34+ hematopoietic stem and progenitor cells as target cells for somatic gene therapy, the current state of FA gene therapy trials, and future prospects for cell and gene therapy. (C) 2009 Elsevier B.V. All rights reserved.

引用

页码：141 / 149

页数：9

共 106 条

[101] Spontaneous functional correction of homozygous Fanconi anaemia alleles reveals novel mechanistic basis for reverse mosaicism [J].