DISCLAIMER
The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.compose your message
message sent
email sent successfully
Trusted Resources: News & Events
Latest announcements and gatherings
Step toward gene therapy for sickle cell disease
A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.
The team went on to demonstrate that the mended cells could make a functioning hemoglobin molecule, which carries oxygen in normal red blood cells, and then successfully transplanted the stem cells into mice. The researchers say the study represents a proof of concept for the repair of blood-borne genetic diseases, such as sickle cell disease and thalassemia.
Related Content
-
news & eventsRandomized Trials Needed to Assess Benefits of Salmonella Vaccines in SCD Patients, Study SaysRandomized controlled trials are needed ...
-
education & researchChanges in transcranial doppler flow velocities in children with sickle cell disease: The impact of hydroxyurea ther...Background and Objectives: Hydroxyurea ...
-
news & eventsMount Sinai receives NIH grant to study use of inhaled corticosteroids for sickle cell treatmentThe Departments of Emergency Medicine an...
-
news & eventsFDA Approves Novel Treatment to Target Abnormality in Sickle Cell DiseaseToday, the U.S. Food and Drug Administra...
-
news & eventsBioverativ and Bicycle Therapeutics Enter into Strategic Research Collaboration to Develop Therapies for Hemophilia ...Bioverativ Inc. (NASDAQ: BIVV), a global...
-
news & eventsbluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Depende...First patient with severe sickle cell di...
-
videos & visualsWhat is a bone marrow stem cell transplant?http://www.sicklecelltransplantconsortiu...
send a message
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.
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.