Open Advanced Search

Log in
Enterprise Plans
Sign Up

Log in
Sign Up
Enterprise Plans
Browse
Features
Pricing

DeepDyve

Advanced Search

show/hide query preview

With all of the words or DOIs:

DeepDyve's default query mode: search by keyword or DOI.

With the exact phrase:

Require these words, in this exact order. Do not surround your terms in double-quotes ("") in this field.

With at least one of the words:

Find any of these words, separated by spaces

Without the words:

Exclude each of these words, separated by spaces

With words in the article title:

Search for these terms only in the title of an article

Published between the years:

With the following authors:

Most effective as: LastName, First Name or Lastname, FN

With words in the journal name:

Search for articles published in journals where these words are in the journal name

Reset filters

Get 20M+ Full-Text Papers For Less Than $1.50/day. Start a 14-Day Trial for You or Your Team.

Learn More →

DeepDyve requires Javascript to function. Please enable Javascript on your browser to continue.

Gene and Cell Therapies for Beta-Globinopathies

Part of the Advances in Experimental Medicine and Biology Book Series (volume 1013)

Editors: Malik, Punam; Tisdale, John

DOI: 10.1007/978-1-4939-7299-9

ISBN: 978-1-4939-7297-5

Publisher: Springer New York - Springer Journals

Nov 11, 2017

1 - 9 of 9 Chapters

LitStream

Chapter 1Clinical Features of β-Thalassemia and Sickle Cell Disease

McGann, Patrick T.; Nero, Alecia C.; Ware, Russell E.

Pages: 1 –26

[Sickle cell disease (SCD) and β-thalassemia are among the most common inherited diseases, affecting millions of persons globally. It is estimated that 5–7% of the world’s population is a carrier of a significant hemoglobin variant. Without early diagnosis followed by initiation of preventative...
LitStream

Chapter 2Genetic Basis and Genetic Modifiers of β-Thalassemia and Sickle Cell Disease

Thein, Swee Lay

Pages: 27 –57

[β-thalassemia and sickle cell disease (SCD) are prototypical Mendelian single gene disorders, both caused by mutations affecting the adult β-globin gene. Despite the apparent genetic simplicity, both disorders display a remarkable spectrum of phenotypic severity and share two major genetic...
LitStream

Chapter 3Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease

Chonat, Satheesh; Quinn, Charles T.

Pages: 59 –87

[Thalassemia and sickle cell disease (SCD) are disorders of hemoglobin that affect millions of people worldwide. The carrier states for these diseases arose as common, balanced polymorphisms during human history because they afforded protection against severe forms of malaria. These complex,...
LitStream

Chapter 4Allogeneic/Matched Related Transplantation for β-Thalassemia and Sickle Cell Anemia

Bernaudin, Françoise; Pondarré, Corinne; Galambrun, Claire; Thuret, Isabelle

Pages: 89 –122

[Allogeneic hematopoietic stem cell transplantation (HSCT) can cure single gene disorders such as thalassemia and sickle cell anemia (SCA). These non-malignant diseases have in common severe hemolytic anemia and high proliferative bone marrow, requiring frequent transfusions. The risk of...
LitStream

Chapter 5Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease

Fitzhugh, Courtney D.; Abraham, Allistair; Hsieh, Matthew M.

Pages: 123 –153

[Considerable progress with respect to donor source has been achieved in allogeneic stem cell transplant for patients with hemoglobin disorders, with matched sibling donors in the 1980s, matched unrelated donors and cord blood sources in the 1990s, and haploidentical donors in the 2000s. Many...
LitStream

Chapter 6Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia

Dong, Alisa C.; Rivella, Stefano

Pages: 155 –176

[Beta-thalassemia and sickle cell anemia are two of the most common diseases related to the hemoglobin protein. In these diseases, the beta-globin gene is mutated, causing severe anemia and ineffective erythropoiesis. Patients can additionally present with a number of life-threatening...
LitStream

Chapter 7Reactivation of Fetal Hemoglobin for Treating β-Thalassemia and Sickle Cell Disease

Cui, Shuaiying; Engel, James Douglas

Pages: 177 –202

[Reactivation of fetal hemoglobin (HbF) in adult hematopoietic cells has the potential for great clinical benefit in patients bearing deleterious mutations in the β-globin gene, such as β-thalassemia and sickle cell disease (SCD), since increasing the production of HbF can compensate for...
LitStream

Chapter 8Genome Editing for the β-Hemoglobinopathies

Porteus, Matthew H.

Pages: 203 –217

[The β-hemoglobinopathiesβ-hemoglobinopathiesHBB geneβ-globin (HBB) gene are diverse set of disorders caused by mutations in the β-globin (HBB) gene. Because HBB proteinβ-globin (HBB) geneHBB protein is a critical component (along with α-globin, heme, and iron) of hemoglobin, the molecule...
LitStream

Chapter 9Gene and Cell Therapy for β-Thalassemia and Sickle Cell Disease with Induced Pluripotent Stem Cells (iPSCs): The Next Frontier

Papapetrou, Eirini P.

Pages: 219 –240

[In recent years, breakthroughs in human pluripotent stem cell (hPSC) research, namely cellular reprogramming and the emergence of sophisticated genetic engineering technologies, have opened new frontiers for cell and gene therapy. The prospect of using hPSCs, either autologous or...

results per page

Browse All eBooks

Features

Search and discover articles on DeepDyve, PubMed, and Google Scholar
Read the full-text of open access and premium content
Organize articles with folders and bookmarks
Collaborate on and share articles and folders

Info

Pricing
Enterprise Plans
Browse Journals & Topics
About DeepDyve

Help

Help
Publishers
Contact Us

Sign up for your DeepDyve account now!

Read and print from thousands of top scholarly journals.

System error. Please try again!

Continue with Facebook

By signing up, you agree to DeepDyve’s Terms of Service and Privacy Policy.

Already have an account? Log in

Save Article to Bookmarks

Bookmark this article. You can see your Bookmarks on your DeepDyve Library.

To save an article, log in first, or sign up for a DeepDyve account if you don’t already have one.

Subscribe to Journal Email Alerts

To subscribe to email alerts, please log in first, or sign up for a DeepDyve account if you don’t already have one.

Follow a Journal

To get new article updates from a journal on your personalized homepage, please log in first, or sign up for a DeepDyve account if you don’t already have one.

Our policy towards the use of cookies

All DeepDyve websites use cookies to improve your online experience. They were placed on your computer when you launched this website. You can change your cookie settings through your browser.

cookie policy