Abstract
Background: Induced pluripotent stem cells hold tremendous potential for biological and therapeutic applications. The development of efficient technologies for targeted genome alteration of stem cells in disease models is a prerequisite for utilizing stem cells to their full potential. The revolutionary technology for genome editing known as the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein 9 (Cas9) system is recently recognized as a powerful tool for editing DNA at specific loci.
Objective: The ease of use of the CRISPR-Cas9 technology will allow us to improve our understanding of genomic variation in disease processes via cellular and animal models. More recently, this system was modified to repress (CRISPR interference, CRISPRi) or activate (CRISPR activation, CRISPRa) gene expression without alterations in the DNA, which amplified the scope of applications of CRISPR systems for stem cell biology.
Results and Conclusion: Here, we highlight latest advances of CRISPR-associated applications in human pluripotent stem cells. The challenges and future prospects of CRISPR-based systems for human research are also discussed.
Keywords: CRISPR/Cas9, CRISPRi, CRISPRa, pluripotent stem cells, iPSC, therapy.
Current Stem Cell Research & Therapy
Title:CRISPR/Cas9 Genome Editing: A Promising Tool for Therapeutic Applications of Induced Pluripotent Stem Cells
Volume: 13 Issue: 4
Author(s): Yanli Zhang, Danuta Sastre and Feng Wang*
Affiliation:
- College of Animal Science, Nanjing Agricultural University, Nanjing,China
Keywords: CRISPR/Cas9, CRISPRi, CRISPRa, pluripotent stem cells, iPSC, therapy.
Abstract: Background: Induced pluripotent stem cells hold tremendous potential for biological and therapeutic applications. The development of efficient technologies for targeted genome alteration of stem cells in disease models is a prerequisite for utilizing stem cells to their full potential. The revolutionary technology for genome editing known as the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein 9 (Cas9) system is recently recognized as a powerful tool for editing DNA at specific loci.
Objective: The ease of use of the CRISPR-Cas9 technology will allow us to improve our understanding of genomic variation in disease processes via cellular and animal models. More recently, this system was modified to repress (CRISPR interference, CRISPRi) or activate (CRISPR activation, CRISPRa) gene expression without alterations in the DNA, which amplified the scope of applications of CRISPR systems for stem cell biology.
Results and Conclusion: Here, we highlight latest advances of CRISPR-associated applications in human pluripotent stem cells. The challenges and future prospects of CRISPR-based systems for human research are also discussed.
Export Options
About this article
Cite this article as:
Zhang Yanli , Sastre Danuta and Wang Feng *, CRISPR/Cas9 Genome Editing: A Promising Tool for Therapeutic Applications of Induced Pluripotent Stem Cells, Current Stem Cell Research & Therapy 2018; 13 (4) . https://dx.doi.org/10.2174/1574888X13666180214124800
DOI https://dx.doi.org/10.2174/1574888X13666180214124800 |
Print ISSN 1574-888X |
Publisher Name Bentham Science Publisher |
Online ISSN 2212-3946 |
- 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
-
Ethical Implications for Clinical Practice and Future Research in “At Risk” Individuals
Current Pharmaceutical Design Immunotherapeutic Approaches in MS: Update on Pathophysiology and Emerging Agents or Strategies 2006
Endocrine, Metabolic & Immune Disorders - Drug Targets The Crystal Structures of <i>Thermomyces (Humicola) Lanuginosa</i> Lipase in Complex with Enzymatic Reactants
Current Enzyme Inhibition The Interaction Between Burn Injury and Vitamin D Metabolism and Consequences for the Patient
Current Clinical Pharmacology Age-related Vascular Differences among Patients Suffering from Multiple Sclerosis
Current Neurovascular Research New Insights into the Biology of 1-Antitrypsin and its Role in Chronic Obstructive Pulmonary Disease
Current Respiratory Medicine Reviews Potential for Stem Cells Therapy in Alzheimer’s Disease: Do Neurotrophic Factors Play Critical Role?
Current Alzheimer Research Biobanks between Common Good and Private Interest: The Example of Umbilical Cord Blood Private Biobanks
Recent Patents on DNA & Gene Sequences Preclinical Models of Multiple Sclerosis: Advantages and Limitations Towards Better Therapies
Current Medicinal Chemistry Neuronal Cell Death in Alzheimers Disease and a Neuroprotective Factor, Humanin
Current Neuropharmacology Biomarkers, Biosensors and Biomedicine
Current Medicinal Chemistry Inhibitors of the Hedgehog Signal Transduction Pathway
Current Cancer Therapy Reviews Delta Opioid Peptides Derived from Plant Proteins
Current Pharmaceutical Design Microglia-Neuron Interaction in Inflammatory and Degenerative Diseases: Role of Cholinergic and Noradrenergic Systems
CNS & Neurological Disorders - Drug Targets 5-HT2A Inverse-Agonists for the Treatment of Insomnia
Current Topics in Medicinal Chemistry Receptor Heteromers in Parkinson’s Disease and L-DOPA-Induced Dyskinesia
CNS & Neurological Disorders - Drug Targets Transdermal Drug Delivery: Opportunities and Challenges for Controlled Delivery of Therapeutic Agents Using Nanocarriers
Current Drug Metabolism Molecular Self-Assembly of Peptide Nanostructures: Mechanism of Association and Potential Uses
Current Nanoscience Methionine-Independent Translation Initiation from Naturally Occurring Non-AUG Codons
Current Chemical Biology The Role of Levomilnacipran in the Management of Major Depressive Disorder: A Comprehensive Review
Current Neuropharmacology