Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Gene Therapy: An Experimental Technique to Treat Genetic Diseases
source: Sickle Cell Anemia News
year: N/A
summary/abstract:Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administration approved the first gene therapy for an inherited disease.
Gene Therapy for Sickle Cell Anemia
Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.
Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patient’s own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.
read more
+myBinderRelated Content
-
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approachesSickle cell disease is an inherited diso...
-
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell DiseaseBackground: Sickle cell disease is char...
-
MaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative r...MaxCyte, Inc. today announces it has e...
-
bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Depende...First patient with severe sickle cell di...
-
‘Natural’ Gene Mutation May Offer Way of Treating Sickle Cell Disease, Study SaysIntroducing a “natural” genetic muta...
-
Sickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
-
Boston Children’s Hospital receives grant for sickle cell disease researchThe Bill and Melinda Gates Foundation aw...
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.