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Titlebook: Gene and Cell Therapies for Beta-Globinopathies; Punam Malik,John Tisdale Book 2017 Springer Science+Business Media LLC 2017 Beta-Globinop

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,Gene and Cell Therapy for β-Thalassemia and Sickle Cell Disease with Induced Pluripotent Stem Cellsc 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 medici
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Book 2017 which are often available only to 15-20% of patients. An alternative to allogeneic HS.CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally,
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,Clinical Features of β-Thalassemia and Sickle Cell Disease,ive treatment for SCD but less so for β-thalassemia, and does not represent curative therapy. As technology and use of cellular and gene therapies expand, SCD and thalassemia should be among the highest disease priorities.
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,Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease,some of the unrelated cord transplant studies. Haploidentical donors have emerged in the last decade to have less GvHD; however, improvements are needed to increase the engraftment rate. Thus the decision to use unrelated cord blood units or haploidentical donors may depend on the institutional expe
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