Keratinocyte gene therapy.
Keratinocyte gene therapy.
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角质形成细胞基因治疗。
DOI：
10.1001/archderm.1993.01680320112015
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发表时间：
1993
影响因子：
--
通讯作者：
Jonathan C. Vogel
中科院分区：
文献类型：
--
作者：
Jonathan C. Vogel
背景
基因疗法目前正在临床试验中用于治疗多种疾病。在角质形成细胞基因治疗中，将通过表达正常蛋白质或酶来纠正疾病的基因插入角质形成细胞中并在角质形成细胞中表达。角质形成细胞作为基因治疗的靶细胞，在治疗全身性疾病以及由角质形成细胞遗传缺陷引起的皮肤病方面具有显著的潜力。
意见
虽然角质形成细胞基因疗法尚未在临床试验中进行测试，但确实存在动物模型，其中角质形成细胞被用于分泌因子如人生长激素和因子IX（用于血友病）进入体循环。皮肤的遗传性疾病，如隐性营养不良性大疱性表皮病或着色性干皮病尚未在动物模型中用角质形成细胞基因疗法治疗。
结论
角质形成细胞作为基因治疗的靶细胞具有许多优点，利用动物模型已经取得了进展。然而，通过表达转基因的角质形成细胞将因子持续有效地递送至血流尚未实现。未来的目标是在角质形成细胞或血管系统中持续获得足够水平的所需因子、激素或酶。
BACKGROUND
Gene therapy is currently being used in clinical trials to treat a variety of diseases. In keratinocyte gene therapy, the gene that will correct the disease by expressing the normal protein or enzyme is inserted and expressed in keratinocytes. Keratinocytes have significant potential, as a target cell of gene therapy, in the treatment of both systemic diseases as well as skin diseases caused by a genetic defect in keratinocytes.
OBSERVATIONS
Although keratinocyte gene therapy is not yet being tested in clinical trials, animal models do exist where keratinocytes are being used to secrete factors such as human growth hormone and factor IX (for hemophilia) into the systemic circulation. Genetic diseases of the skin such as recessive epidermolysis bullosa dystrophica or xeroderma pigmentosum have not yet been treated with keratinocyte gene therapy in animal models.
CONCLUSIONS
Keratinocytes have many advantages as a target cell in gene therapy, and progress has been made using animal models. However, the sustained and efficient delivery of factors to the bloodstream by keratinocytes expressing a transgene has not yet been accomplished. Future goals are to obtain adequate levels of the desired factors, hormones, or enzymes for sustained periods of time, either in keratinocytes or in the vascular system.
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血友病犬皮肤成纤维细胞中因子 IX 缺乏的表型校正。
DOI：
10.1073/pnas.87.13.5173
发表时间：
1990
影响因子：
11.1
作者：
Axelrod,JH;Read,MS;Brinkhous,KM;Verma,IM
通讯作者：
Verma,IM
影响因子：
10.5
作者：
LEASK, A;ROSENBERG, M;FUCHS, E
通讯作者：
FUCHS, E
转基因小鼠表皮过度表达转化生长因子β1抑制皮肤发育。
DOI：
10.1073/pnas.90.11.5237
发表时间：
1993
影响因子：
11.1
作者：
Sellheyer,K;Bickenbach,JR;Rothnagel,JA;Bundman,D;Longley,MA;Krieg,T;Roche,NS;Roberts,AB;Roop,DR
通讯作者：
Roop,DR
移植后，狗皮肤成纤维细胞中的高水平人腺苷脱氨酶表达不会持续。
影响因子：
4.2
作者：
Ramesh,N;Lau,S;Palmer,TD;Storb,R;Osborne,WR
通讯作者：
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通过转基因皮肤成纤维细胞在动物体内产生人类因子 IX：B 型血友病的潜在疗法。
影响因子：
20.3
作者：
Palmer,TD;Thompson,AR;Miller,AD
通讯作者：
Miller,AD