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
-
Notch Signaling in Leukemias and Lymphomas
Current Molecular Medicine Interactions of Metal Ions with DNA, Its Constituents and Derivatives, which may be Relevant for Anticancer Research
Current Topics in Medicinal Chemistry A Systematic Review of Selected Musculoskeletal Late Effects in Survivors of Childhood Cancer
Current Pediatric Reviews Inhibitors of Cancer Stem Cells
Anti-Cancer Agents in Medicinal Chemistry Targeting of Peptide Cytotoxins to LHRH Receptors For Treatment of Cancer
Current Drug Targets Acetylenic Anticancer Agents
Anti-Cancer Agents in Medicinal Chemistry Delivery Methods of Camptothecin and Its Hydrosoluble Analogue Irinotecan for Treatment of Colorectal Cancer
Current Drug Delivery Recent Advances in the Therapeutic Perspectives of Nutlin-3
Current Pharmaceutical Design Cullin-RING Ligases as Attractive Anti-cancer Targets
Current Pharmaceutical Design Liposomes: An Emerging Approach for the Treatment of Cancer
Current Pharmaceutical Design Recent Studies on Macromolecular Synthesis Labeled with 3H-Thymidine in Various Organs as Revealed by Electron Microscopic Radioautography
Current Radiopharmaceuticals Molecular Mechanisms of Drug Photodegradation and Photosensitization
Current Pharmaceutical Design Preventive and Therapeutic Effects of the Retinoid X Receptor Agonist Bexarotene on Tumors
Current Drug Metabolism MicroRNAs in Human Virus Genomes: Helping Hands for Viral Infection
MicroRNA Primary Cardiac Tumors: A Retrospective Study
Current Respiratory Medicine Reviews Oncogene-Blocking Therapies: New Insights from Conditional Mouse Tumor Models
Current Cancer Drug Targets Src Kinase Inhibitors: An Update on Patented Compounds
Current Medicinal Chemistry Evaluation of Non-Coding RNAs as Potential Targets in Head and Neck Squamous Cell Carcinoma Cancer Stem Cells
Current Drug Targets The Application of Fungal Beta-glucans for the Treatment of Colon Cancer
Anti-Cancer Agents in Medicinal Chemistry The Renin Angiotensin System in the Regulation of Angiogenesis
Current Pharmaceutical Design