In situ (in vivo) gene transfer into murine bone marrow stem cells.

In situ (in vivo) gene transfer into murine bone marrow stem cells.
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原位(体内)基因转移至小鼠骨髓干细胞中。

DOI:
10.1007/978-1-59745-409-4_12
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发表时间:
2009
期刊:
Methods in molecular biology (Clifton, N.J.)
影响因子:
--
通讯作者:
Pan,Dao
Pan,Dao
中科院分区:
--
文献类型:
--
作者:
Pan,Dao

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成人骨髓干细胞是基因治疗遗传性疾病、某些恶性肿瘤和艾滋病的理想靶细胞。通过股内(IF)注射的慢病毒载体(LV)介导的干细胞基因转移的体内方法可以充分利用驻留在骨腔中的任何干细胞来源。这种方法可以避免离体造血干细胞(HSC)基因转移遇到的几个困难。我们已经表明,HSC和间充质干/祖细胞(MSC)可以成功地通过一个单一的“原位”IF注射在其自然的“小生境”在小鼠中没有任何预处理的遗传修饰。这种方法可能为治疗人类疾病提供一种新的应用,并代表了一种有趣的新工具来研究成体干细胞的可塑性和不受干扰的造血的性质。
Adult bone marrow stem cell is an ideal target for gene therapy of genetic diseases, selected malignant diseases, and AIDS. The in vivo approach of lentivirus vector (LV)-mediated stem cell gene transfer by intrafemoral (IF) injection can take full advantage of any source of stem cells residing in the bone cavity. Such an approach may avoid several difficulties encountered by ex vivo hematopoietic stem cell (HSC) gene transfer. We have shown that both HSC and mesenchymal stem/progenitor cells (MSC) can be genetically modified successfully by a single “in situ” IF injection in their natural “niche” in mice without any preconditioning. This approach may provide a novel application for treatment of human diseases, and represent an interesting new tool to study adult stem cell plasticity and the nature of unperturbed hematopoiesis.
通过直接股骨内注射进行体内逆转录病毒基因转移可纠正 Jak3 敲除动物的 SCID 表型。
DOI: 10.1182/blood-2002-12-3859
发表时间: 2003
期刊: Blood
影响因子: 20.3
作者:
McCauslin,ChristineS;Wine,John;Cheng,Linzhao;Klarmann,KimD;Candotti,Fabio;Clausen,PeterA;Spence,SallyE;Keller,JonathanR
通讯作者: Keller,JonathanR
通过慢病毒载体的原位递送将基因体内基因转移到未条件化小鼠的成体干细胞中。
DOI: 10.1016/j.ymthe.2006.05.014
发表时间: 2006
期刊: Molecular therapy : the journal of the American Society of Gene Therapy
影响因子: --
作者:
Worsham,DNicole;Schuesler,Todd;vonKalle,Christof;Pan,Dao
通讯作者: Pan,Dao
DOI: 10.1006/mthe.2002.0630
发表时间: 2002-07-01
期刊: MOLECULAR THERAPY
影响因子: 12.4
作者:
Pan, D;Gunther, R;Whitley, CB
通讯作者: Whitley, CB