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
-
Cancer “Stemness”- Regulating MicroRNAs: Role, Mechanisms and Therapeutic Potential
Current Drug Targets Editorial:
Pharmaceutical Nanotechnology Tyrosine Kinase Inhibitors
Current Cancer Drug Targets Antibody-Drug Conjugate Targets
Current Cancer Drug Targets PET Radiopharmaceuticals for Personalized Medicine
Current Drug Targets Cancer and Cyclooxygenase-2 (COX-2) Inhibition
Current Pharmaceutical Design Current Status and Future Prospects of C1 Domain Ligands as Drug Candidates
Current Topics in Medicinal Chemistry Retraction Notice: Protein Identification in Sub Proteome Fractions of Breast Cancer Cells by OFFGEL-IEF and iTRAQ Labeling
Current Proteomics Targeting Telomerase for Cancer Therapy
Current Cancer Therapy Reviews Antimicrobial Peptides in Oral Cancer
Current Pharmaceutical Design Differences in Phosphorylated Histone H2AX Foci Formation and Removal of Cells Exposed to Low and High Linear Energy Transfer Radiation
Current Genomics Recent Developments in the Field of Anticancer Platinum Complexes
Recent Patents on Anti-Cancer Drug Discovery Targeting Ras-RAF-ERK and its Interactive Pathways as a Novel Therapy for Malignant Gliomas
Current Cancer Drug Targets Is the Clinical Use of Cannabis by Oncology Patients Advisable?
Current Medicinal Chemistry Immunomodulatory Lactoferrin in the Regulation of Apoptosis Modulatory Proteins in Cancer
Protein & Peptide Letters Natural Products as Promising Antitumoral Agents in Breast Cancer: Mechanisms of Action and Molecular Targets.
Mini-Reviews in Medicinal Chemistry Manipulation of Glycolysis in Malignant Tumors: Fantasy or Therapy?
Current Medicinal Chemistry Different Aspects of Head and Neck Squamous Cell Carcinoma: Cancer Stem Cells, their Niche and Targeted Therapy
Current Stem Cell Research & Therapy Selection and Characterization of Human Anti-MAGE-A1 scFv and Immunotoxin
Anti-Cancer Agents in Medicinal Chemistry Adenovirus-Based Cancer Gene Therapy
Current Gene Therapy