Gene and Cell Therapies for Beta-Globinopathies / Nejlevnější knihy
Gene and Cell Therapies for Beta-Globinopathies

Kód: 16402079

Gene and Cell Therapies for Beta-Globinopathies

Autor Md Malik, Ph. D. Tisdale

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 o ... celý popis

5060


Skladem u dodavatele
Odesíláme za 14-18 dnů
Přidat mezi přání

Mohlo by se vám také líbit

Dárkový poukaz: Radost zaručena

Objednat dárkový poukazVíce informací

Více informací o knize Gene and Cell Therapies for Beta-Globinopathies

Nákupem získáte 506 bodů

Anotace knihy

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, 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.

Parametry knihy

Zařazení knihy Knihy v angličtině Medicine Pre-clinical medicine: basic sciences Medical genetics

5060

Oblíbené z jiného soudku



Osobní odběr Praha, Brno a 12903 dalších

Copyright ©2008-24 nejlevnejsi-knihy.cz Všechna práva vyhrazenaSoukromíCookies


Můj účet: Přihlásit se
Všechny knihy světa na jednom místě. Navíc za skvělé ceny.

Nákupní košík ( prázdný )

Vyzvednutí v Zásilkovně
zdarma nad 1 499 Kč.

Nacházíte se: