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Literature DB >> 30340033

Stem Cells, Genome Editing, and the Path to Translational Medicine.

Abstract

The derivation of human embryonic stem cells (hESCs) and the stunning discovery that somatic cells can be reprogrammed into human induced pluripotent stem cells (hiPSCs) holds the promise to revolutionize biomedical research and regenerative medicine. In this Review, we focus on disorders of the central nervous system and explore how advances in human pluripotent stem cells (hPSCs) coincide with evolutions in genome engineering and genomic technologies to provide realistic opportunities to tackle some of the most devastating complex disorders.

Entities: Chemical Disease Gene Species

Mesh：

Year: 2018 PMID： 30340033 PMCID： PMC6461399 DOI： 10.1016/j.cell.2018.09.010

Source DB: PubMed Journal: Cell ISSN： 0092-8674 Impact factor: 41.582

172 in total

1. Genetic heterogeneity in human disease.

Authors: Jon McClellan; Mary-Claire King
Journal: Cell Date: 2010-04-16 Impact factor: 41.582

2. Derivation of novel human ground state naive pluripotent stem cells.

Authors: Ohad Gafni; Leehee Weinberger; Abed AlFatah Mansour; Yair S Manor; Elad Chomsky; Dalit Ben-Yosef; Yael Kalma; Sergey Viukov; Itay Maza; Asaf Zviran; Yoach Rais; Zohar Shipony; Zohar Mukamel; Vladislav Krupalnik; Mirie Zerbib; Shay Geula; Inbal Caspi; Dan Schneir; Tamar Shwartz; Shlomit Gilad; Daniela Amann-Zalcenstein; Sima Benjamin; Ido Amit; Amos Tanay; Rada Massarwa; Noa Novershtern; Jacob H Hanna
Journal: Nature Date: 2013-10-30 Impact factor: 49.962

3. Highly efficient endogenous human gene correction using designed zinc-finger nucleases.

Authors: Fyodor D Urnov; Jeffrey C Miller; Ya-Li Lee; Christian M Beausejour; Jeremy M Rock; Sheldon Augustus; Andrew C Jamieson; Matthew H Porteus; Philip D Gregory; Michael C Holmes
Journal: Nature Date: 2005-04-03 Impact factor: 49.962

4. ApoE2, ApoE3, and ApoE4 Differentially Stimulate APP Transcription and Aβ Secretion.

Authors: Yu-Wen Alvin Huang; Bo Zhou; Marius Wernig; Thomas C Südhof
Journal: Cell Date: 2017-01-19 Impact factor: 41.582

5. Breaking the code of DNA binding specificity of TAL-type III effectors.

Authors: Jens Boch; Heidi Scholze; Sebastian Schornack; Angelika Landgraf; Simone Hahn; Sabine Kay; Thomas Lahaye; Anja Nickstadt; Ulla Bonas
Journal: Science Date: 2009-12-11 Impact factor: 47.728

6. Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9.

Authors: Dominik Paquet; Dylan Kwart; Antonia Chen; Andrew Sproul; Samson Jacob; Shaun Teo; Kimberly Moore Olsen; Andrew Gregg; Scott Noggle; Marc Tessier-Lavigne
Journal: Nature Date: 2016-04-27 Impact factor: 49.962

7. Discovery and validation of sub-threshold genome-wide association study loci using epigenomic signatures.

Authors: Xinchen Wang; Nathan R Tucker; Gizem Rizki; Robert Mills; Peter Hl Krijger; Elzo de Wit; Vidya Subramanian; Eric Bartell; Xinh-Xinh Nguyen; Jiangchuan Ye; Jordan Leyton-Mange; Elena V Dolmatova; Pim van der Harst; Wouter de Laat; Patrick T Ellinor; Christopher Newton-Cheh; David J Milan; Manolis Kellis; Laurie A Boyer
Journal: Elife Date: 2016-05-10 Impact factor: 8.140

8. Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes.

Authors:
Journal: Cell Stem Cell Date: 2012-06-28 Impact factor: 24.633

9. Human cerebral organoids recapitulate gene expression programs of fetal neocortex development.

Authors: J Gray Camp; Farhath Badsha; Marta Florio; Sabina Kanton; Tobias Gerber; Michaela Wilsch-Bräuninger; Eric Lewitus; Alex Sykes; Wulf Hevers; Madeline Lancaster; Juergen A Knoblich; Robert Lachmann; Svante Pääbo; Wieland B Huttner; Barbara Treutlein
Journal: Proc Natl Acad Sci U S A Date: 2015-12-07 Impact factor: 11.205

10. Human Brain Organoids on a Chip Reveal the Physics of Folding.

Authors: Eyal Karzbrun; Aditya Kshirsagar; Sidney R Cohen; Jacob H Hanna; Orly Reiner
Journal: Nat Phys Date: 2018-02-19 Impact factor: 20.034

43 in total

Review 1. Genetics of extreme human longevity to guide drug discovery for healthy ageing.

Authors: Zhengdong D Zhang; Sofiya Milman; Jhih-Rong Lin; Shayne Wierbowski; Haiyuan Yu; Nir Barzilai; Vera Gorbunova; Warren C Ladiges; Laura J Niedernhofer; Yousin Suh; Paul D Robbins; Jan Vijg
Journal: Nat Metab Date: 2020-07-27

Review 2. New frontiers in modeling tuberous sclerosis with human stem cell-derived neurons and brain organoids.

Authors: John D Blair; Helen S Bateup
Journal: Dev Dyn Date: 2019-05-23 Impact factor: 3.780

Review 3. Genetic predispositions of Parkinson's disease revealed in patient-derived brain cells.

Authors: Jenne Tran; Helena Anastacio; Cedric Bardy
Journal: NPJ Parkinsons Dis Date: 2020-04-24

4. Engineered Heart Muscle Models in Phenotypic Drug Screens.

Authors: Wolfram-Hubertus Zimmermann
Journal: Handb Exp Pharmacol Date: 2021

Review 5. Autophagy Dysfunction as a Phenotypic Readout in hiPSC-Derived Neuronal Cell Models of Neurodegenerative Diseases.

Authors: Congxin Sun; Tatiana R Rosenstock; Malkiel A Cohen; Sovan Sarkar
Journal: Methods Mol Biol Date: 2022

6. Formation of Human Neuroblastoma in Mouse-Human Neural Crest Chimeras.

Authors: Malkiel A Cohen; Shupei Zhang; Satyaki Sengupta; Haiting Ma; George W Bell; Brendan Horton; Bandana Sharma; Rani E George; Stefani Spranger; Rudolf Jaenisch
Journal: Cell Stem Cell Date: 2020-03-05 Impact factor: 24.633

7. Functional analysis of CX3CR1 in human induced pluripotent stem (iPS) cell-derived microglia-like cells.

Authors: Nobuhito Murai; Maisam Mitalipova; Rudolf Jaenisch
Journal: Eur J Neurosci Date: 2020-09-14 Impact factor: 3.386

8. Intravital imaging of mouse embryos.

Authors: Qiang Huang; Malkiel A Cohen; Fernando C Alsina; Garth Devlin; Aliesha Garrett; Jennifer McKey; Patrick Havlik; Nikolai Rakhilin; Ergang Wang; Kun Xiang; Parker Mathews; Lihua Wang; Cheryl Bock; Victor Ruthig; Yi Wang; Marcos Negrete; Chi Wut Wong; Preetish K L Murthy; Shupei Zhang; Andrea R Daniel; David G Kirsch; Yubin Kang; Blanche Capel; Aravind Asokan; Debra L Silver; Rudolf Jaenisch; Xiling Shen
Journal: Science Date: 2020-04-10 Impact factor: 47.728

9. Notch signaling induces either apoptosis or cell fate change in multiciliated cells during mucociliary tissue remodeling.

Authors: Alexia Tasca; Martin Helmstädter; Magdalena Maria Brislinger; Maximilian Haas; Brian Mitchell; Peter Walentek
Journal: Dev Cell Date: 2021-01-04 Impact factor: 12.270

Review 10. Therapeutic genome editing in cardiovascular diseases.

Authors: Masataka Nishiga; Lei S Qi; Joseph C Wu
Journal: Adv Drug Deliv Rev Date: 2020-02-21 Impact factor: 15.470