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
-
Dasatinib in the Treatment of Chronic Myeloid Leukemia
Current Signal Transduction Therapy Active-Targeted Nanotherapy Strategies for Prostate Cancer
Current Cancer Drug Targets Recent Patents Therapeutic Agents for Cancer
Recent Patents on Anti-Cancer Drug Discovery Detection of Predictive Markers for Therapeutic Stratification of Salivary Glands Tumors
Current Drug Targets Cytoplasmic CXCR4 High-Expression Exhibits Distinct Poor Clinicopathological Characteristics and Predicts Poor Prognosis in Triple-Negative Breast Cancer
Current Molecular Medicine Lanthanides as Anticancer Agents
Current Medicinal Chemistry - Anti-Cancer Agents Plant Secondary Metabolites in Cancer Chemotherapy: Where are We?
Current Pharmaceutical Biotechnology Baseline CD4/CD8 T-Cell Ratio Predicts Prompt Immune Restoration Upon cART Initiation
Current HIV Research mTOR Inhibition and the Tumor Vasculature
Current Angiogenesis (Discontinued) Human Chorionic Gonadotropin: A Model Molecule For Oligopeptide-Based Drug Discovery
Endocrine, Metabolic & Immune Disorders - Drug Targets Peptides to Target Tumor Vasculature and Lymphatics for Improved Anti-Angiogenesis Therapy
Current Cancer Drug Targets Bioactive Secondary Metabolites from Endophytic Fungi
Current Medicinal Chemistry An Overview of the Chemistry and Pharmacological Potentials of Furanones Skeletons
Current Organic Chemistry Drug Combinatorial Therapies for the Treatment of KRAS Mutated Lung Cancers
Current Topics in Medicinal Chemistry Perspectives in Biomolecular Therapeutic Intervention in Cancer: From the Early to the New Strategies With Type I Interferons
Current Medicinal Chemistry New Development and Application of Ultrasound Targeted Microbubble Destruction in Gene Therapy and Drug Delivery
Current Gene Therapy Innovative Cancer Treatments that Augment Radiotherapy or Chemotherapy by the Use of Immunotherapy or Gene Therapy
Recent Patents on Anti-Cancer Drug Discovery Extracellular Tropomyosin: A Novel Common Pathway Target for Anti- Angiogenic Therapy
Current Cancer Drug Targets Polymeric Carriers for Gene Delivery: Chitosan and Poly(amidoamine) Dendrimers
Current Pharmaceutical Design Radiosynthesis of [18F]-fluorobenzoate-doxorubicin Using Acylation Approach
Current Radiopharmaceuticals