Can I read Gene and Cell Therapies for Beta-Globinopathies Volume 1013 on EtoBox?

Gene and Cell Therapies for Beta-Globinopathies Volume 1013 by Malik, Punam; Tisdale, John is a scholarly article available to read on EtoBox.

What is Gene and Cell Therapies for Beta-Globinopathies Volume 1013 about?

Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 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 manipulation of the defective organ might be equally therapeutic. However, procedural toxicities and the requirement of an HLA-matched sibling donor limit this approach to a fraction of affected individuals. The editors review the progress & the state of the field in HSCT for hemoglobinopathies & shed light on the major changes expected in the next decade. Although allogeneic HSCT is a curative option, it is limited by the availability of matched donors, 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,

Author

Malik, Punam; Tisdale, John

Publisher

Springer New York : Imprint : Springer

Published

2017

Language

EN

ISBN

9781493972975