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: Community Center
Online groups, photo galleries and blogs
3 Gene Editing Approaches for Sickle Cell Disease
Sickle cell disease (SCD) is a perfect candidate for gene editing. It is perhaps the best understood single-gene condition, due to a substitution of a single DNA base in the gene that encodes the beta subunit of hemoglobin, the protein that carries oxygen in the blood.
Red blood cells, which bend into the sickle shape in the disease, obstructing circulation and causing excruciating pain, descend from hematopoietic (“blood-forming”) stem cells (HSCs) in the bone marrow. So theoretically, a patient’s stem cells can be removed, the mutation replaced in them, and then the fixed cells infused back into the patient, circumventing an immune response.
+myBinderRelated Content
-
news & eventsMaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative r...MaxCyte, Inc. today announces it has e...
-
education & researchHABIT, a Randomized Feasibility Trial to Increase Hydroxyurea Adherence, Suggests Improved Health Related Quality of...Objectives: To examine the effect of a...
-
education & researchTranscutaneous electrical nerve stimulation (TENS) for pain management in sickle cell diseaseAs defined by the American Physical Ther...
-
Community CenterWhat it’s like to be 17 and living with sickle cell diseaseTre Adegoroye is 17 and was diagnosed wi...
-
news & eventsGhana Launches Partnership With Novartis to Improve Diagnosis and Treatment of People With Sickle Cell DiseaseThe Government of Ghana announced a new ...
-
news & eventsSickle cell patients in UK survey, especially those 16 to 20, voice problems with care and pain relief givenIn a survey, patients across the...
-
videos & visualsAlabama Man Free of Sickle Cell After Gene Therapyhttps://www.youtube.com/watch?v=E9vYON8D...
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.