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狗模型在开发基因治疗策略以促进人类健康方面的作用。

The function of dog models in developing gene therapy strategies for human health.

机构信息

Department of Genetics and Biochemistry, Clemson University, SC 29634-0318, USA.

出版信息

Mamm Genome. 2011 Aug;22(7-8):476-85. doi: 10.1007/s00335-011-9348-0. Epub 2011 Jul 6.

DOI:10.1007/s00335-011-9348-0
PMID:21732191
Abstract

The domestic dog is of great benefit to humankind, not only through companionship and working activities cultivated through domestication and selective breeding, but also as a model for biomedical research. Many single-gene traits have been well-characterized at the genomic level, and recent advances in whole-genome association studies will allow for better understanding of complex, multigenic hereditary diseases. Additionally, the dog serves as an invaluable large animal model for assessment of novel therapeutic agents. Thus, the dog has filled a crucial step in the translation of basic research to new treatment regimens for various human diseases. Four well-characterized diseases in canine models are discussed as they relate to other animal model availability, novel therapeutic approach, and extrapolation to human gene therapy trials.

摘要

家犬对人类有很大的好处,不仅通过驯化和选择性繁殖培养了陪伴和工作活动,而且还作为生物医学研究的模型。许多单基因性状已经在基因组水平上得到了很好的描述,全基因组关联研究的最新进展将有助于更好地理解复杂的多基因遗传性疾病。此外,狗作为一种非常有价值的大型动物模型,可用于评估新型治疗药物。因此,狗在将基础研究转化为治疗各种人类疾病的新疗法方面发挥了至关重要的作用。本文将讨论犬模型中四种特征明确的疾病,因为它们与其他动物模型的可用性、新的治疗方法以及对人类基因治疗试验的推断有关。

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Cytokine-enhanced vaccine and interferon-β plus suicide gene as combined therapy for spontaneous canine sarcomas.细胞因子增强疫苗和干扰素-β联合自杀基因治疗自发性犬肉瘤。
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Mammalian models of Duchenne Muscular Dystrophy: pathological characteristics and therapeutic applications.杜兴氏肌肉营养不良症的哺乳动物模型:病理特征与治疗应用
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Treatment of canine leukocyte adhesion deficiency by foamy virus vectors expressing CD18 from a PGK promoter.
犬黑色素瘤的比较研究
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Genome-wide association study to identify potential genetic modifiers in a canine model for Duchenne muscular dystrophy.全基因组关联研究,以确定杜兴氏肌营养不良犬模型中的潜在基因修饰因子。
BMC Genomics. 2016 Aug 22;17(1):665. doi: 10.1186/s12864-016-2948-z.
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Resiniferatoxin: The Evolution of the "Molecular Scalpel" for Chronic Pain Relief.树脂毒素:用于缓解慢性疼痛的“分子手术刀”的演变
Pharmaceuticals (Basel). 2016 Aug 11;9(3):47. doi: 10.3390/ph9030047.
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Gene therapy in monogenic congenital myopathies.单基因先天性肌病的基因治疗
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Comparative Genomics of X-linked Muscular Dystrophies: The Golden Retriever Model.X 连锁肌营养不良症的比较基因组学:金毛猎犬模型。
Curr Genomics. 2013 Aug;14(5):330-42. doi: 10.2174/13892029113149990004.
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Isolation, genetic manipulation, and transplantation of canine spermatogonial stem cells: progress toward transgenesis through the male germ-line.犬精原干细胞的分离、遗传操作和移植:通过雄性生殖系进行转基因的进展。
Reproduction. 2013 Jun 14;146(1):75-90. doi: 10.1530/REP-13-0086. Print 2013 Jul.
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Mol Ther. 2011 Feb;19(2):251-9. doi: 10.1038/mt.2010.265. Epub 2010 Dec 7.
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Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression.利用携带人源 CD11b 和 CD18 启动子驱动犬源 CD18 表达的慢病毒载体对犬白细胞黏附缺陷症进行基因治疗。
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Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies.在黑暗中点燃蜡烛:隐性视网膜营养不良的遗传学和基因治疗进展。
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