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用于模拟人类疾病的转基因猪。

Genetically modified pigs to model human diseases.

作者信息

Flisikowska Tatiana, Kind Alexander, Schnieke Angelika

出版信息

J Appl Genet. 2014 Feb;55(1):53-64. doi: 10.1007/s13353-013-0182-9.

DOI:10.1007/s13353-013-0182-9
PMID:24234401
Abstract

Genetically modified mice are powerful tools to investigate the molecular basis of many human diseases. Mice are, however, of limited value for preclinical studies, because they differ significantly from humans in size, general physiology, anatomy and lifespan. Considerable efforts are, thus, being made to develop alternative animal models for a range of human diseases. These promise powerful new resources that will aid the development of new diagnostics, medicines and medical procedures. Here, we provide a comprehensive review of genetically modified porcine models described in the scientific literature: various cancers, cystic fibrosis, Duchenne muscular dystrophy, autosomal polycystic kidney disease, Huntington’s disease, spinal muscular atrophy, haemophilia A, X-linked severe combined immunodeficiency, retinitis pigmentosa, Stargardt disease, Alzheimer’s disease, various forms of diabetes mellitus and cardiovascular diseases.

摘要

转基因小鼠是研究许多人类疾病分子基础的有力工具。然而,小鼠在临床前研究中的价值有限,因为它们在体型、一般生理学、解剖学和寿命方面与人类有显著差异。因此,人们正在付出巨大努力来开发针对一系列人类疾病的替代动物模型。这些有望成为强大的新资源,有助于新诊断方法、药物和医疗程序的开发。在这里,我们对科学文献中描述的转基因猪模型进行了全面综述:各种癌症、囊性纤维化、杜氏肌营养不良症、常染色体显性多囊肾病、亨廷顿舞蹈症、脊髓性肌萎缩症、甲型血友病、X连锁重症联合免疫缺陷病、色素性视网膜炎、斯特格氏病、阿尔茨海默病、各种形式的糖尿病和心血管疾病。

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J Appl Genet. 2014 Feb;55(1):53-64. doi: 10.1007/s13353-013-0182-9.
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本文引用的文献

1
A transgenic minipig model of Huntington's Disease.亨廷顿舞蹈症的转基因小型猪模型。
J Huntingtons Dis. 2013;2(1):47-68. doi: 10.3233/JHD-130001.
2
Efficient nonmeiotic allele introgression in livestock using custom endonucleases.利用定制内切酶实现家畜中非减数分裂等位基因的高效导入。
Proc Natl Acad Sci U S A. 2013 Oct 8;110(41):16526-31. doi: 10.1073/pnas.1310478110. Epub 2013 Sep 6.
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Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle.肌营养不良症猪为肌营养不良症肌肉生理紊乱的层次结构提供了新的见解。
使用小鼠异种移植肝脏模型进行用于转化猪研究的腺相关病毒衣壳筛选
bioRxiv. 2024 May 29:2024.05.29.596409. doi: 10.1101/2024.05.29.596409.
4
Genome Editing in Pigs.猪的基因组编辑。
Methods Mol Biol. 2023;2631:393-417. doi: 10.1007/978-1-0716-2990-1_19.
5
Inhibitory Effects of 3-Methylcholanthrene Exposure on Porcine Oocyte Maturation.3-甲基胆蒽暴露对猪卵母细胞成熟的抑制作用。
Int J Mol Sci. 2023 Mar 14;24(6):5567. doi: 10.3390/ijms24065567.
6
Application of the transgenic pig model in biomedical research: A review.转基因猪模型在生物医学研究中的应用:综述。
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Profiling development of abdominal organs in the pig.猪的腹部器官发育分析。
Sci Rep. 2022 Sep 28;12(1):16245. doi: 10.1038/s41598-022-19960-5.
8
A humanized minipig model for the toxicological testing of therapeutic recombinant antibodies.用于治疗性重组抗体毒理学测试的人源化小型猪模型。
Nat Biomed Eng. 2022 Nov;6(11):1248-1256. doi: 10.1038/s41551-022-00921-2. Epub 2022 Sep 22.
9
Judgment Bias During Gestation in Domestic Pigs.家猪妊娠期的判断偏差
Front Vet Sci. 2022 May 12;9:881101. doi: 10.3389/fvets.2022.881101. eCollection 2022.
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MSC therapy in livestock models.家畜模型中的间充质干细胞疗法。
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Hum Mol Genet. 2013 Nov 1;22(21):4368-82. doi: 10.1093/hmg/ddt287. Epub 2013 Jun 19.
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Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs.肠型 CFTR 的表达缓解了囊性纤维化猪的胎粪性肠梗阻。
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Generation of c-Myc transgenic pigs for autosomal dominant polycystic kidney disease.生成用于常染色体显性多囊肾病的 c-Myc 转基因猪。
Transgenic Res. 2013 Dec;22(6):1231-9. doi: 10.1007/s11248-013-9707-6. Epub 2013 Mar 30.
6
RNA guides genome engineering.RNA引导基因组工程。
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7
Genomic responses in mouse models poorly mimic human inflammatory diseases.小鼠模型中的基因组反应与人类炎症性疾病的反应相差很大。
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8
CFTR-deficient pigs display peripheral nervous system defects at birth.CFTR 缺陷型猪在出生时就表现出外周神经系统缺陷。
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