Abstract
Induced pluripotent stem cells (iPSCs) represent an invaluable tool in a chromosomal instability syndrome such as Fanconi anemia (FA), as they can allow to study of the molecular defects underlying this disease. Many other applications, such as its use as a platform to test different methods or compounds, could also be of interest. But the greatest impact of iPSCs may be in bone marrow failure diseases, as iPSCs could represent an unlimited source of autologous cells to apply in advanced treatments such as gene therapy. At the same time, genome editing constitutes the next generation of technology to further develop a safer, personalized, targeted gene therapy. Despite the promising advantages that these two technologies would present in a disease such as FA, the specific characteristics of the disease make both of these processes especially challenging. Efficient and safer FA-hiPSC (human induced pluripotent stem cell) generation methods, robust and reliable differentiation protocols for iPSCs, as well as really efficient delivery methods to perform targeted gene correction should be developed.
Keywords: Induced pluripotent stem cells, Gene editing, Fanconi anemia, Gene therapy.
Current Gene Therapy
Title:Induced Pluripotency and Gene Editing in Fanconi Anemia
Volume: 16 Issue: 5
Author(s): Susana Navarro, Alessandra Giorgetti, Angel Raya and Jakub Tolar
Affiliation:
Keywords: Induced pluripotent stem cells, Gene editing, Fanconi anemia, Gene therapy.
Abstract: Induced pluripotent stem cells (iPSCs) represent an invaluable tool in a chromosomal instability syndrome such as Fanconi anemia (FA), as they can allow to study of the molecular defects underlying this disease. Many other applications, such as its use as a platform to test different methods or compounds, could also be of interest. But the greatest impact of iPSCs may be in bone marrow failure diseases, as iPSCs could represent an unlimited source of autologous cells to apply in advanced treatments such as gene therapy. At the same time, genome editing constitutes the next generation of technology to further develop a safer, personalized, targeted gene therapy. Despite the promising advantages that these two technologies would present in a disease such as FA, the specific characteristics of the disease make both of these processes especially challenging. Efficient and safer FA-hiPSC (human induced pluripotent stem cell) generation methods, robust and reliable differentiation protocols for iPSCs, as well as really efficient delivery methods to perform targeted gene correction should be developed.
Export Options
About this article
Cite this article as:
Navarro Susana, Giorgetti Alessandra, Raya Angel and Tolar Jakub, Induced Pluripotency and Gene Editing in Fanconi Anemia, Current Gene Therapy 2016; 16 (5) . https://dx.doi.org/10.2174/1566523217666170118112050
DOI https://dx.doi.org/10.2174/1566523217666170118112050 |
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
-
Pharmacological Modulation of Nitric Oxide Release: New Pharmacological Perspectives, Potential Benefits and Risks
Current Medicinal Chemistry Inflammation and Cancer: When NF-κB Amalgamates the Perilous Partnership
Current Cancer Drug Targets Cancer Gene Therapy Utilizing Interleukin-13 Receptor α2 Chain
Current Gene Therapy Molecular Imaging with Small Animal PET/CT
Current Medical Imaging Cardiovascular Remodeling Induced by Passive Smoking
Inflammation & Allergy - Drug Targets (Discontinued) New Entrants into Clinical Trials for Targeted Therapy of Breast Cancer: An Insight
Anti-Cancer Agents in Medicinal Chemistry Clients and Oncogenic Roles of Molecular Chaperone gp96/grp94
Current Topics in Medicinal Chemistry Noscapine and its Analogs as Chemotherapeutic Agent: Current updates
Current Topics in Medicinal Chemistry Targeting ErbB3: the New RTK(id) on the Prostate Cancer Block
Immunology, Endocrine & Metabolic Agents in Medicinal Chemistry (Discontinued) Surgical Management of Medullary Thyroid Carcinoma in Pediatric Age
Current Pediatric Reviews The Role of Mammalian Target of Rapamycin (mTOR) Inhibitors in the Treatment of Solid Tumors
Current Cancer Therapy Reviews Different 6-Aryl-Fulvenes Exert Anti-proliferative effects on Cancer Cells
Anti-Cancer Agents in Medicinal Chemistry Breast Cancer Diagnosis Using Data Mining Methods, Cumulative Histogram Features, and Gary Level Co-occurrence Matrix
Current Medical Imaging PI3K/Akt/mTOR Pathway Inhibitors in Cancer: A Perspective on Clinical Progress
Current Medicinal Chemistry The Role of Tregs in Cancer: Foxp3 as a Putative Target for Therapy
Current Signal Transduction Therapy Epidemiology of Candida albicans Infections and Role of Non-Candidaalbicans Yeasts
Current Drug Targets TEM8 Targeted Cancer Therapy
Anti-Cancer Agents in Medicinal Chemistry Neuropharmacology of the Endocannabinoid Signaling System-Molecular Mechanisms, Biological Actions and Synaptic Plasticity
Current Neuropharmacology Control of Protein Synthesis in Malignant Transformation - the Role of eIF4E and the eIF4E Binding Proteins in the Regulation of Apoptosis
Current Cancer Therapy Reviews Redistribution of CD95 into the Lipid Rafts to Treat Cancer Cells?
Recent Patents on Anti-Cancer Drug Discovery