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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Clinical Epidemiology and Biostatisticsions in the β-globin gene, such as β-thalassemia and sickle cell disease (SCD), since increasing the production of HbF can compensate for underproduction of β-globin chains (in β-thalassemia) and it can also disrupt sickle hemoglobin polymerization (in SCD). Thus for the past few decades, concerted

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Hepatitis B Virus: Asian Perspective, with α-globin, heme, and iron) of hemoglobin, the molecule essential for oxygen delivery to tissues, mutations in . can result in lethal diseases or diseases with multi-organ dysfunction. . mutations can be roughly divided into two categories: those that cause a dysfunctional protein (such as sickl

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phase-2-enzyme

Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease,tem diseases are reviewed here with a focus on current standards of clinical management and recent research findings. The importance of a comprehensive, multidisciplinary and lifelong system of care is also emphasized.

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Book 2017early 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipula

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Luke B. Hesson,Antonia L. Pritchard 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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