Genetic Correction and Hepatic Differentiation of Hemophilia B-specific Human Induced Pluripotent Stem Cells.
Reads0
Chats0
TLDR
Mutations in human disease-specific iPSCs could be precisely corrected by CRISPR/Cas9 technology, and corrected cells still maintained hepatic differentiation capability, and the findings might throw a light on iPSC-based personalized therapies in the clinical application.About:
This article is published in Chinese Medical Sciences Journal.The article was published on 2017-09-01 and is currently open access. It has received 10 citations till now. The article focuses on the topics: Point mutation & Induced pluripotent stem cell.read more
Citations
More filters
Journal ArticleDOI
Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease.
Martijn P.T. Ernst,Mike Broeders,Pablo Herrero-Hernandez,Esmee Oussoren,Ans T. van der Ploeg,W.W.M. Pim Pijnappel +5 more
TL;DR: An overview of clinical trials involving gene editing using clustered interspaced short palindromic repeats (CRISPR)- CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), or zinc finger nuclease (ZFNs) is presented and the underlying mechanisms are discussed.
Journal ArticleDOI
Development and application of CRISPR/Cas9 technologies in genomic editing
Cui Zhang,Renfu Quan,Jinfu Wang +2 more
TL;DR: The application of the CRISPR system over the last 2 years is reviewed, including its development and application in base editing, transcription modulation and epigenetic editing, genomic-scale screening, and cell and embryo therapy.
Journal ArticleDOI
Differentiation of human induced pluripotent stem cells into erythroid cells
Mohsen Ebrahimi,Mehdi Forouzesh,Setareh Raoufi,Mohammad Ramazii,Farhoodeh Ghaedrahmati,Maryam Farzaneh +5 more
TL;DR: The in vitro production of human iPSC-derived RBCs can be an alternative treatment option for patients with blood disorders and an overview of the current status and applications of this field is presented.
Journal ArticleDOI
Recent Updates on Induced Pluripotent Stem Cells in Hematological Disorders
TL;DR: An overview of induced pluripotent stem cells, patient-specific iPSCs for disease modeling and drug screening, applications of iPSC and genome editing technology in hematological disorders, remaining challenges, and future perspectives ofiPSCs in heMatological diseases will be discussed.
Journal ArticleDOI
Current approaches in CRISPR-Cas9 mediated gene editing for biomedical and therapeutic applications.
Gargi Bhattacharjee,N. M. Gohil,Khushal Khambhati,Indra Mani,Rupesh Maurya,Janardhan Keshav Karapurkar,J H Gohil,Dinh-Toi Chu,Hue Vu Thi,K. Alzahrani,Pau Loke Show,Rakesh Rawal,S. Ramakrishna,Vijai Singh +13 more
TL;DR: In this paper , the authors highlight recent developments and utilization of the CRISPR-Cas9 system in correcting or generating gene mutations to create model organisms to develop deeper insights into diseases, rescue normal gene functionality, and curb the progression of a disease.
References
More filters
Journal ArticleDOI
Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors.
TL;DR: Induction of pluripotent stem cells from mouse embryonic or adult fibroblasts by introducing four factors, Oct3/4, Sox2, c-Myc, and Klf4, under ES cell culture conditions is demonstrated and iPS cells, designated iPS, exhibit the morphology and growth properties of ES cells and express ES cell marker genes.
Journal ArticleDOI
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.
Martin Jinek,Krzysztof Chylinski,Krzysztof Chylinski,Ines Fonfara,Michael H. Hauer,Jennifer A. Doudna,Emmanuelle Charpentier +6 more
TL;DR: This study reveals a family of endonucleases that use dual-RNAs for site-specific DNA cleavage and highlights the potential to exploit the system for RNA-programmable genome editing.
Journal ArticleDOI
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,Le Cong,F. Ann Ran,F. Ann Ran,David M. Cox,David M. Cox,Shuailiang Lin,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +15 more
TL;DR: The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage as discussed by the authors.
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,F. A. Ran,David Benjamin Turitz Cox,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +10 more
TL;DR: Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
Journal ArticleDOI
Genome engineering using the CRISPR-Cas9 system
F. Ann Ran,Patrick D. Hsu,Jason Wright,Vineeta Agarwala,Vineeta Agarwala,David A. Scott,Feng Zhang +6 more
TL;DR: A set of tools for Cas9-mediated genome editing via nonhomologous end joining (NHEJ) or homology-directed repair (HDR) in mammalian cells, as well as generation of modified cell lines for downstream functional studies are described.