Chapter title |
Gene and Cell Therapy for β-Thalassemia and Sickle Cell Disease with Induced Pluripotent Stem Cells (iPSCs): The Next Frontier
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Chapter number | 9 |
Book title |
Gene and Cell Therapies for Beta-Globinopathies
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Published in |
Advances in experimental medicine and biology, November 2017
|
DOI | 10.1007/978-1-4939-7299-9_9 |
Pubmed ID | |
Book ISBNs |
978-1-4939-7297-5, 978-1-4939-7299-9
|
Authors |
Papapetrou, Eirini P., Eirini P. Papapetrou |
Abstract |
In recent years, breakthroughs in human pluripotent stem cell (hPSC) research, namely cellular reprogramming and the emergence of sophisticated genetic engineering technologies, have opened new frontiers for cell and gene therapy. The prospect of using hPSCs, either autologous or histocompatible, as targets of genetic modification and their differentiated progeny as cell products for transplantation, presents a new paradigm of regenerative medicine of potential tremendous value for the treatment of blood disorders, including beta-thalassemia (BT) and sickle cell disease (SCD). Despite advances at a remarkable pace and great promise, many roadblocks remain before clinical translation can be realistically considered. Here we discuss the theoretical advantages of cell therapies utilizing hPSC derivatives, recent proof-of-principle studies and the main challenges towards realizing the potential of hPSC therapies in the clinic. |
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Mendeley readers
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Unknown | 25 | 100% |
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Student > Bachelor | 5 | 20% |
Other | 2 | 8% |
Student > Master | 2 | 8% |
Lecturer | 1 | 4% |
Professor | 1 | 4% |
Other | 3 | 12% |
Unknown | 11 | 44% |
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Biochemistry, Genetics and Molecular Biology | 9 | 36% |
Medicine and Dentistry | 3 | 12% |
Pharmacology, Toxicology and Pharmaceutical Science | 1 | 4% |
Arts and Humanities | 1 | 4% |
Unknown | 11 | 44% |