CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells
TL;DR: The progress made in correcting gene mutations in monogenic hereditary disorders is highlighted and various CRISPR-associated applications, such as cancer research, synthetic biology, and gene therapy using induced pluripotent stem cells are discussed.
read more
Abstract: Recent advances in genome editing with programmable nucleases have opened up new avenues for multiple applications, from basic research to clinical therapy. The ease of use of the technology-and particularly clustered regularly interspaced short palindromic repeats (CRISPR)-will allow us to improve our understanding of genomic variation in disease processes via cellular and animal models. Here, we highlight the progress made in correcting gene mutations in monogenic hereditary disorders and discuss various CRISPR-associated applications, such as cancer research, synthetic biology, and gene therapy using induced pluripotent stem cells. The challenges, ethical issues, and future prospects of CRISPR-based systems for human research are also discussed.
read more
Chat with Paper
AI Agents for this Paper
Find similar papers on Google Scholar, PubMed and Arxiv
Write a critical review of this paper
Analyze citations of this paper to find unaddressed research gaps
Citations
Therapeutic genome editing: prospects and challenges
David Benjamin Turitz Cox,Randall Jeffrey Platt,Feng Zhang +2 more
- 01 Feb 2015
TL;DR: Current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges are discussed.
964
Cancer-derived exosomes as a delivery platform of CRISPR/Cas9 confer cancer cell tropism-dependent targeting
TL;DR: It is reported that cancer‐derived exosomes function as natural carriers that can efficiently deliver CRISPR/Cas9 plasmids to cancer through selective accumulation in ovarian cancer tumors of SKOV3 xenograft mice.
388
Disease Modeling Using 3D Organoids Derived from Human Induced Pluripotent Stem Cells.
TL;DR: Studying human diseases using hiPSC-derived organoids may better illustrate the processes involved due to similarities in the architecture and microenvironment present in an organoid, which also allows drug responses to be properly recapitulated in vitro.
156
CRISPR/Cas9-based targeted genome editing for correction of recessive dystrophic epidermolysis bullosa using iPS cells.
Joanna Jacków,Zongyou Guo,Corey Hansen,Hasan E. Abaci,Y. Doucet,J. Shin,R. Hayashi,D. Delorenzo,Y. Kabata,Satoru Shinkuma,Julio C. Salas-Alanis,Angela M. Christiano +11 more
TL;DR: It is demonstrated that the combination of iPSCs, gene editing, and tissue engineering allows us to perform efficient gene correction by homology-directed repair with the CRISPR-Cas9 gRNA ribonucleoprotein complex system, and generate human skin equivalents that show skin integrity and type VII collagen restoration after grafting them onto nude mice.
104
References
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.
16.2K
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
- 01 Feb 2013
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.
12K
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali,Luhan Yang,Kevin M. Esvelt,John Aach,Marc Güell,James E. DiCarlo,Julie E. Norville,George M. Church,George M. Church +8 more
TL;DR: The type II bacterial CRISPR system is engineer to function with custom guide RNA (gRNA) in human cells to establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering.
The new frontier of genome engineering with CRISPR-Cas9
TL;DR: The power of the CRISPR-Cas9 technology to systematically analyze gene functions in mammalian cells, study genomic rearrangements and the progression of cancers or other diseases, and potentially correct genetic mutations responsible for inherited disorders is illustrated.
6.1K