Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease
source: Blood
year: 2016
authors: Hoban MD, Orkin SH, Bauer DE
summary/abstract:Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent and severe monogenic disorder, SCD has been long considered a logical candidate for gene therapy. Significant progress has been made in moving toward this goal. These efforts have provided substantial insight into the natural regulation of the globin genes and illuminated challenges for genetic manipulation of the hematopoietic system. The initial γ-retroviral vectors, next-generation lentiviral vectors, and novel genome engineering and gene regulation approaches each share the goal of preventing erythrocyte sickling. After years of preclinical studies, several clinical trials for SCD gene therapies are now open. This review focuses on progress made toward achieving gene therapy, the current state of the field, consideration of factors that may determine clinical success, and prospects for future development.
organization: Boston Children's Hospital; Dana-Farber Cancer Institute, Boston; Harvard Medical School, Harvard Stem Cell Institute, BostonDOI: 10.1182/blood-2015-09-618587
read more full text
+myBinderRelated Content
-
FDA Awards UNC Researcher $2M Grant to Study Kidney Disease in Sickle Cell AnemiaThe U.S. Food and Drug Administration (F...
-
A Validated Measure of Adherence to Antibiotic Prophylaxis in Children With Sickle Cell DiseaseAntibiotic prophylaxis is a mainstay in ...
-
Older Blood Used in Transfusions May Be Harmful to Adult Sickle Cell Patients, Review FindsIn an analysis of adults with sickle cel...
-
The Experience and Health-Related Quality of Life After Haploidentical Stem Cell Transplantation for Adults With Sic...Haploidentical hematopoietic stem cell t...
-
Nashville Doctor Performs First Successful Gene Editing Procedure on Sickle Cell Anemia Patienthttps://www.youtube.com/watch?v=s7TPRomV...
-
ASH and FDA Unveil New Recommendations to Guide Clinical Development of Sickle Cell Disease TherapiesThe American Society of Hematology (ASH)...
-
SCDAA News Advisory: Partial Hold on Gene Therapy TrialOn December 20, the FDA placed a partial...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.