Human iPSCs and Genome Editing Technologies for Precision Cardiovascular Tissue Engineering.
Human iPSCs and Genome Editing Technologies for Precision Cardiovascular Tissue Engineering.
复制标题
DOI：
10.3389/fcell.2021.639699
复制
发表时间：
2021
影响因子：
5.5
通讯作者：
Emmert MY
中科院分区：
文献类型：
--
作者：
Gähwiler EKN;Motta SE;Martin M;Nugraha B;Hoerstrup SP;Emmert MY
诱导多能干细胞(IPSCs)起源于使用四种Yamanaka转录因子对成体细胞进行重编程。自他们发现以来，干细胞(SC)领域取得了重大里程碑，并在疾病建模、药物发现和再生医学领域开辟了几个大门。与此同时，簇状规则间隔短回文重复序列(CRISPR)相关蛋白9(CRISPR-Cas9)的出现彻底改变了基因组工程领域，使转基因细胞系的产生成为可能，并实现了精确的基因组重组或随机插入/缺失，这对更广泛的应用是有用的。心血管疾病代表着一个不断增加的社会关注，对潜在的细胞和分子机制了解有限。IPSCs分化为多种细胞类型的能力与CRISPR-Cas9技术相结合，可能使系统研究病理生理机制或筛选潜在治疗药物成为可能。此外，这些技术可以通过调节靶蛋白的表达或抑制为心血管组织工程(TE)方法提供细胞平台，从而为设计新的细胞系和/或微调仿生支架创造了可能性。本文就IPSCs、CRISPR-Cas9及其组合在心血管血栓栓塞症领域的应用作一综述。特别是，将讨论这些技术的临床可翻译性，范围从疾病建模到药物筛选和TE应用。
Induced pluripotent stem cells (iPSCs) originate from the reprogramming of adult somatic cells using four Yamanaka transcription factors. Since their discovery, the stem cell (SC) field achieved significant milestones and opened several gateways in the area of disease modeling, drug discovery, and regenerative medicine. In parallel, the emergence of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (CRISPR-Cas9) revolutionized the field of genome engineering, allowing the generation of genetically modified cell lines and achieving a precise genome recombination or random insertions/deletions, usefully translated for wider applications. Cardiovascular diseases represent a constantly increasing societal concern, with limited understanding of the underlying cellular and molecular mechanisms. The ability of iPSCs to differentiate into multiple cell types combined with CRISPR-Cas9 technology could enable the systematic investigation of pathophysiological mechanisms or drug screening for potential therapeutics. Furthermore, these technologies can provide a cellular platform for cardiovascular tissue engineering (TE) approaches by modulating the expression or inhibition of targeted proteins, thereby creating the possibility to engineer new cell lines and/or fine-tune biomimetic scaffolds. This review will focus on the application of iPSCs, CRISPR-Cas9, and a combination thereof to the field of cardiovascular TE. In particular, the clinical translatability of such technologies will be discussed ranging from disease modeling to drug screening and TE applications.
登录
查看更多内容
人类3D心脏微动物的表征和验证，以评估心脏病理变化。
影响因子：
4.6
作者：
Archer CR;Sargeant R;Basak J;Pilling J;Barnes JR;Pointon A
通讯作者：
Pointon A
影响因子：
64.8
作者：
Anders, Carolin;Niewoehner, Ole;Duerst, Alessia;Jinek, Martin
通讯作者：
Jinek, Martin
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：
20.1
作者：
Bezzina, Connie R.;Lahrouchi, Najim;Priori, Silvia G.
通讯作者：
Priori, Silvia G.
影响因子：
--
作者：
Abrams, D J;Perkin, M A;Skinner, J R
通讯作者：
Skinner, J R