Punam Malik, MD; John Tisdale, Ph.D. Gene and Cell Therapies for Beta-Globinopathies 9781493972975

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Автор: Punam Malik, MD; John Tisdale, Ph.D.
Название: Gene and Cell Therapies for Beta-Globinopathies
ISBN: 9781493972975
Издательство: Springer
Классификация:

Медицинская генетика

Клеточная биология (цитология)

ISBN-10: 1493972979
Обложка/Формат: Hardcover
Страницы: 433
Вес: 0.55 кг.
Дата издания: 20.11.2017
Серия: Advances in experimental medicine and biology
Язык: English
Издание: 1st ed. 2017
Иллюстрации: 12 illustrations, color; 4 illustrations, black and white; xii, 248 p. 16 illus., 12 illus. in color.
Размер: 163 x 244 x 23
Читательская аудитория: Professional & vocational
Основная тема: Biomedicine
Ссылка на Издательство: Link
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Поставляется из: Германии
Описание:

Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the worlds 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, targeted genetic correction is emerging as a novel therapeutic strategy in the hemoglobinopathies. Although ideal, the inefficiency of targeted correction was rate limiting for translation of this technology to the clinic. With advancements in zinc finger nucleases and TALE endonuclease mediated targeted correction, correction frequencies in hematopoietic stem cells is now reaching levels that may become clinically relevant. Furthermore, the ability to generate autologous embryonic stem cell like cells from primary somatic cells (skin fibroblasts or hematopoietic cells) of the affected individual has allowed for the potential application of genetic correction strategies.This Book reviews upcoming genetic strategies to reactivate fetal hemoglobin production and research advances.

Gene and Cell Therapies for Beta-Globinopathies, Punam Malik, MD; John Tisdale, Ph.D.