Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View

被引：25

作者：

Cavazzana, Marina ^{[1
,2
,3
,4
]}

Ribeil, Jean-Antoine ^{[1
]}

Lagresle-Peyrou, Chantal ^{[2
,3
,4
]}

Andre-Schmutz, Isabelle ^{[2
,3
,4
]}

机构：

[1] Necker Childrens Hosp, AP HP, Biotherapy Dept, F-75015 Paris, France

[2] INSERM, Grp Hosp Univ Ouest, AP HP, Biotherapy Clin Invest Ctr, Paris, France

[3] Paris Descartes Sorbonne Paris Cite Univ, Imagine Inst, Paris, France

[4] INSERM, UMR 1163, Lab Human Lymphohematopoiesis, Paris, France

来源：

STEM CELLS AND DEVELOPMENT | 2017年 / 26卷 / 02期

基金：

欧洲研究理事会; 欧盟地平线“2020”;

关键词：

bone marrow; gene therapy; HSC; WISKOTT-ALDRICH-SYNDROME; CHRONIC GRANULOMATOUS-DISEASE; RED-BLOOD-CELLS; SYNDROME PROTEIN; INEFFECTIVE ERYTHROPOIESIS; BETA-THALASSEMIA; CD34(+) CELLS; ACTIVATION; ANEMIA; IMMUNODEFICIENCY;

D O I：

10.1089/scd.2016.0230

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

When considering inherited diseases that can be treated by gene transfer into hematopoietic stem cells (HSCs), there are only two in which the HSC and progenitor cell distribution inside the bone marrow and its micro-environment are exactly the same as in a healthy subject: metachromatic leukodystrophy (MLD) and adrenoleukodystrophy (ALD). In all other settings [X-linked severe combined immunodeficiency (X-SCID), adenosine deaminase deficiency, Wiskott-Aldrich syndrome, and beta-hemoglobinopathies], the bone marrow content of the different stem and precursor cells and the cells' relationship with the stroma have very specific characteristics. These peculiarities can influence the cells' harvesting and behavior in culture, and the postgraft uptake and further behavior of the gene-modified hematopoietic/precursor cells. In the present mini-review, we shall briefly summarize these characteristics and outline the possible consequences and challenges.

引用

页码：71 / 76

页数：6

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