Google
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
-
Crizanlizumab 5.0 mg/kg increased the time to first on-treatment sickle cell pain crisis: a subgroup analysis of the...In the 52-week SUSTAIN study, which comp...
-
Gamida Cell to Present Data from NiCord® Programs at the 2018 BMT Tandem MeetingsGamida Cell, a leading cellular and immu...
-
2017 SiNERGe Webinar July 14 – Chronic Opioid Therapy & Sickle Cell Diseasehttps://www.youtube.com/watch?v=8WZNTwPD...
-
Diabetes Drug, Metformin, Suggested as ‘Breakthrough’ Treatment for Sickle Cell AnemiaMetformin, a common drug for Type 2 diab...
-
Marijuana Use and Health Behaviors in a US Clinic Sample of Patients With Sickle Cell DiseaseIntroduction: As marijuana use becomes ...
-
Progress in pursuit of sickle cell cureBao, in collaboration with Baylor Colleg...
-
FDA Approves First Targeted Therapy to Treat Patients with Painful Complication of Sickle Cell DiseaseToday, the U.S. Food and Drug Administra...
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.