Application of human induced pluripotent stem cells to model fibrodysplasia ossificans progressiva.
Application of human induced pluripotent stem cells to model fibrodysplasia ossificans progressiva.
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DOI：
10.1016/j.bone.2017.07.003
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发表时间：
2018-04
期刊：
影响因子：
4.1
通讯作者：
Hsiao EC
中科院分区：
文献类型：
--
作者：
Barruet E;Hsiao EC
进行性纤维发育不良(FOP)是一种以大量异位骨化为特征的遗传性疾病。一种骨形态发生蛋白(BMP)受体激活素AI型受体(ACVR1)突变的FOP患者。FOP是一种进行性和衰弱的疾病，其特征是经常在创伤后发生骨形成红斑。由于人诱导多能干细胞(HiPSCs)往往很难或不可能从人类捐赠者那里获得大量的组织，因此它为体外疾病模型的建立和药物筛选提供了一个有吸引力的来源。HiPSC具有自我更新的能力，使我们能够获得大量的起始材料。HiPSCs也有可能分化为体内的任何细胞类型。在这篇综述中，我们讨论了HiPSCs技术在FOP研究中的应用如何改变了我们对FOP疾病发病机制的看法。我们还考虑了在药物发现和再生医学中使用人类IPSCs的持续挑战和新出现的机会。
Fibrodysplasia ossificans progressiva (FOP) is a genetic condition characterized by massive heterotopic ossification. FOP patients with mutations in the Activin A type I receptor (ACVR1), a bone morphogenetic protein (BMP) receptor. FOP is a progressive and debilitating disease characterized by bone formation flares that often occur after trauma. Since it is often difficult or impossible to obtain large amounts of tissue from human donors due to the risks of inciting more heterotopic bone formation, human induced pluripotent stem cells (hiPSCs) provide an attractive source for establishing in vitro disease models and for applications in drug screening. hiPSCs have the ability to self-renew, allowing us to obtain large amounts of starting material. hiPSCs also have the potential to differentiate into any cell types in the body. In this review, we discuss how the application of hiPSCs technology to studying FOP has changed our perspectives on FOP disease pathogenesis. We also consider ongoing challenges and emerging opportunities for the use of human iPSCs in drug discovery and regenerative medicine.
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DOI：
10.1038/nrg3473
发表时间：
2013-06
期刊：
Nature reviews. Genetics
影响因子：
--
作者：
通讯作者：
--
影响因子：
48
作者：
Burridge, Paul W.;Matsa, Elena;Shukla, Praveen;Lin, Ziliang C.;Churko, Jared M.;Ebert, Antje D.;Lan, Feng;Diecke, Sebastian;Huber, Bruno;Mordwinkin, Nicholas M.;Plews, Jordan R.;Abilez, Oscar J.;Cui, Bianxiao;Gold, Joseph D.;Wu, Joseph C.
通讯作者：
Wu, Joseph C.
影响因子：
5.2
作者：
Culbert, Andria L.;Chakkalakal, Salin A.;Theosmy, Edwin G.;Brennan, Tracy A.;Kaplan, Frederick S.;Shore, Eileen M.
通讯作者：
Shore, Eileen M.
影响因子：
17.1
作者：
Hatsell SJ;Idone V;Wolken DM;Huang L;Kim HJ;Wang L;Wen X;Nannuru KC;Jimenez J;Xie L;Das N;Makhoul G;Chernomorsky R;D'Ambrosio D;Corpina RA;Schoenherr CJ;Feeley K;Yu PB;Yancopoulos GD;Murphy AJ;Economides AN
通讯作者：
Economides AN
影响因子：
4.8
作者：
Aerssens, J;Boonen, S;Dequeker, J
通讯作者：
Dequeker, J