In Vivo Hematopoietic Stem Cell Transduction

被引：24

作者：

Richter, Maximilian ^{[1
]}

Stone, Daniel ^{[2
]}

Miao, Carol ^{[3
,4
]}

Humbert, Olivier ^{[5
]}

Kiem, Hans-Peter ^{[5
,6
]}

Papayannopoulou, Thalia ^{[7
]}

Lieber, Andre ^{[1
,8
]}

机构：

[1] Univ Washington, Div Med Genet, 1705 NE Pacific St, Seattle, WA 98195 USA

[2] Fred Hutchinson Canc Res Ctr, Vaccine & Infect Dis Div, 1100 Fairview Ave N, Seattle, WA 98109 USA

[3] Univ Washington, Dept Pediat, 1705 NE Pacific St, Seattle, WA 98195 USA

[4] Seattle Childrens Hosp, Res Inst, Ctr Immun & Immunotherapy, 1900 9th Ave, Seattle, WA 98101 USA

[5] Fred Hutchinson Canc Res Ctr, Div Clin Res, 1100 Fairview Aveune N, Seattle, WA 98109 USA

[6] Univ Washington, Dept Med, Sch Med, 1705 NE Pacific St, Seattle, WA 98195 USA

[7] Univ Washington, Div Hematol, 1705 NE Pacific St, Seattle, WA 98195 USA

[8] Univ Washington, Dept Pathol, 1705 NE Pacific St, Seattle, WA 98195 USA

来源：

HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA | 2017年 / 31卷 / 05期

关键词：

Intravenous; Intraosseal; Viral vectors; Mobilization; SEVERE COMBINED IMMUNODEFICIENCY; ADENOASSOCIATED VIRAL VECTORS; DEPENDENT ADENOVIRAL VECTOR; EFFICIENT GENE-TRANSFER; MURINE HEMOPHILIA-A; FOAMY VIRUS VECTOR; INTRA-BONE MARROW; INTRAVENOUS-INJECTION; PROGENITOR CELLS; INTRAFEMORAL TRANSPLANTATION;

D O I：

10.1016/j.hoc.2017.06.001

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Current protocols for hematopoietic stem cell (HSC) gene therapy, involving the transplantation of ex vivo lentivirus vector-transduced HSCs into myeloablated recipients, are complex and not without risk for the patient. In vivo HSC gene therapy can be achieved by the direct modification of HSCs in the bone marrow after intraosseous injection of gene delivery vectors. A recently developed approach involves the mobilization of HSCs from the bone marrow into the peripheral blood circulation, intravenous vector injection, and re-engraftment of genetically modified HSCs in the bone marrow. We provide examples for in vivo HSC gene therapy and discuss advantages and disadvantages.

引用

页码：771 / +

页数：16

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