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
-
Opioid utilization patterns in United States patients with sickle cell diseaseSickle cell disease (SCD) is the most co...
-
Gene Therapy: What You Need to Know (Warrior FAQs)Two gene therapies were recently approve...
-
Hydroxyurea linked to ‘significant, rapid’ reduction of sperm countSix months of hydroxyurea therapy detrim...
-
Long-term opioids may not be best pain management option for all sickle cell patientsSmall study shows some on opioids report...
-
CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell DiseaseThe California Institute for Regenerativ...
-
Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Diseasehttps://www.youtube.com/watch?v=CWTW5pDP...
-
New Research Suggests SANGUINATE™ Reduces the Number of Sickled Red Blood Cells in Patients with Vaso-Occlusive Cr...SANGUINATE™, the only investigational ...
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.