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 Systematic Analysis of Physicochemical and ADME Properties of All Small Molecule Kinase Inhibitors Approved by US FDA from January 2001 to October 2015
Current Medicinal Chemistry Repurposing of Anticancer Drugs for the Treatment of Bacterial Infections
Current Topics in Medicinal Chemistry Screening of Candidate Pathogenic Genes for Spontaneous Abortion Using Whole Exome Sequencing
Combinatorial Chemistry & High Throughput Screening Lycorine and its Derivatives for Anticancer Drug Design
Mini-Reviews in Medicinal Chemistry Cancer Bioinformatics for Updating Anticancer Drug Developments and Personalized Therapeutics
Reviews on Recent Clinical Trials Kinases as Upstream Regulators of the HIF System: Their Emerging Potential as Anti-Cancer Drug Targets
Current Pharmaceutical Design The Impact of Tumor Physiology on Camptothecin-Based Drug Development
Current Medicinal Chemistry - Anti-Cancer Agents Melphalan in Regional Chemotherapy for Locally Recurrent Metastatic Melanoma
Current Topics in Medicinal Chemistry Tropism-Modified Adenoviral and Adeno-Associated Viral Vectors for Gene Therapy
Current Gene Therapy The Ubiquitin-Proteasome Pathway and Resistance Mechanisms Developed Against the Proteasomal Inhibitors in Cancer Cells
Current Drug Targets Breast Cancer: Current Developments in Molecular Approaches to Diagnosis and Treatment
Recent Patents on Anti-Cancer Drug Discovery Contrast Echocardiography: An Update on Clinical Applications
Current Pharmaceutical Design Camptothecins and Lung Cancer: Improved Delivery Systems by Aerosol
Current Cancer Drug Targets Tumor-Targeted Drug Delivery with Aptamers
Current Medicinal Chemistry Targeting the Immune System in Cancer
Current Pharmaceutical Biotechnology Recent Advances in the Development of Catalytic Inhibitors of Human DNA Topoisomerase IIα As Novel Anticancer Agents
Current Medicinal Chemistry Lentinus edodes: A Macrofungus with Pharmacological Activities
Current Medicinal Chemistry Critical Update and Emerging Trends in Imatinib Treatment for Gastrointestinal Stromal Tumor
Reviews on Recent Clinical Trials Natural Product-Derived Small Molecule Activators of Hypoxia-Inducible Factor-1 (HIF-1)
Current Pharmaceutical Design Mesenchymal Stem Cells: New Approaches for the Treatment of Neurological Diseases
Current Stem Cell Research & Therapy