Abstract
The development of custom-designed nucleases (CDNs), including zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), has made it possible to perform precise genetic engineering in many cell types and species. More recently, clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has been successfully employed for genome editing. These RNA-guided DNA endonucleases are shown to be more efficient and flexible than CDNs and hold great potential for applications in both biological studies and medicine. Here, we review the progress that has been made for all three genome editing technologies in modifying both cells and model organisms, compare important aspects of each approach, and summarize the applications of these tools in disease modeling and gene therapy. In the end, we discuss future prospects of the field.
Keywords: Zinc finger nucleases, TALENs, CRISPR, disease modeling, gene therapy, review.
Current Gene Therapy
Title:Targeted Genome Editing Tools for Disease Modeling and Gene Therapy
Volume: 14 Issue: 1
Author(s): Mi Cai and Yi Yang
Affiliation:
Keywords: Zinc finger nucleases, TALENs, CRISPR, disease modeling, gene therapy, review.
Abstract: The development of custom-designed nucleases (CDNs), including zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), has made it possible to perform precise genetic engineering in many cell types and species. More recently, clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has been successfully employed for genome editing. These RNA-guided DNA endonucleases are shown to be more efficient and flexible than CDNs and hold great potential for applications in both biological studies and medicine. Here, we review the progress that has been made for all three genome editing technologies in modifying both cells and model organisms, compare important aspects of each approach, and summarize the applications of these tools in disease modeling and gene therapy. In the end, we discuss future prospects of the field.
Export Options
About this article
Cite this article as:
Cai Mi and Yang Yi, Targeted Genome Editing Tools for Disease Modeling and Gene Therapy, Current Gene Therapy 2014; 14 (1) . https://dx.doi.org/10.2174/156652321402140318165450
DOI https://dx.doi.org/10.2174/156652321402140318165450 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
A Review of Natural and Modified Betulinic, Ursolic and Echinocystic Acid Derivatives as Potential Antitumor and Anti-HIV Agents
Mini-Reviews in Medicinal Chemistry Role of PARP Inhibitors in Cancer Biology and Therapy
Current Medicinal Chemistry Pharmacology and Therapeutic Applications of Enediyne Antitumor Antibiotics
Current Molecular Pharmacology The Interest of Folic Acid in Targeted Photodynamic Therapy
Current Medicinal Chemistry RNA Interference-Mediated Validation of Survivin and Apollon/BRUCE as New Therapeutic Targets for Cancer Therapy
Current Topics in Medicinal Chemistry Molecular Basis for Invertebrate Innate Immune Recognition of (1→3)-β- D-Glucan as A Pathogen-Associated Molecular Pattern
Current Pharmaceutical Design Antiviral Activities of Human Host Defense Peptides
Current Medicinal Chemistry Agonists and Antagonists of Protease Activated Receptors (PARs)
Current Medicinal Chemistry Inactivated- or Killed-Virus HIV/AIDS Vaccines
Current Drug Targets - Infectious Disorders HDAC as a Therapeutic Target for Treatment of Endometrial Cancers
Current Pharmaceutical Design Mutational Testing in Gastrointestinal Stromal Tumor
Current Cancer Drug Targets 5-Aminoisoquinolin-1-one (5-AIQ), a Water-Soluble Inhibitor of the Poly(ADP-Ribose)Polymerases (PARPs)
Current Medicinal Chemistry Therapeutic Potential of γ -Secretase Inhibitors and Modulators
Current Topics in Medicinal Chemistry Lymphatic Delivery of Anti-HIV Drug Nanoparticles
Recent Patents on Nanotechnology Therapeutic Potential of Endophytic Compounds: A Special Reference to Drug Transporter Inhibitors
Current Topics in Medicinal Chemistry Advances in the Treatment of Ovarian Cancer Using PARP Inhibitors and the Underlying Mechanism of Resistance
Current Drug Targets CXCR4 and Glioblastoma
Anti-Cancer Agents in Medicinal Chemistry Molecular Design and Clinical Development of VEGFR Kinase Inhibitors
Current Topics in Medicinal Chemistry Epidemiology, Clinical Presentation and Treatment of Mucosal Melanoma
Clinical Cancer Drugs Liposomes: A Review of Manufacturing Techniques and Targeting Strategies
Current Nanoscience