Promising results from the first clinical trials of globin gene transfer to treat beta-thalassemias-inherited forms of anemia-have eliminated the need for blood transfusions in some individuals.

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to ...

Please provide your email address to receive an email when new articles are posted on . Thompson described beta-thalassemia as a severe chronic inherited blood disorder due to one of many mutations in ...

Mandy Boontanrart, PhD, a postdoc at ETH Zurich in Switzerland, has a particularly personal motivation for her research on a novel genetic therapy for sickle cell disease (SCD). She is a carrier of a ...

bluebird bio BLUE recently announced that the FDA has approved Zynteglo (betibeglogene autotemcel), as a one-time gene therapy for treating beta-thalassemia, in adult and pediatric patients who ...

Today new and updated data from bluebird bio inc.’ s gene therapy programs in beta-thalassemia and sickle cell disease were presented at the 64 th American Society of Hematology Annual Meeting and ...

SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced the outcome of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory ...

Please provide your email address to receive an email when new articles are posted on . The FDA approved betibeglogene autotemcel for treatment of patients with beta-thalassemia who require routine ...

Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene Therapy. To improve the efficacy of gene therapy when using anti-sickling ...

Thalassemia is a common genetic condition affecting red blood cells. Children with severe forms of thalassemia often develop symptoms early in life, but effective treatment can manage the condition.

Whether you inherit alpha- or beta-thalassemia depends on which genes are affected. The number of affected genes can determine the subtype of alpha- or beta-thalassemia and the severity of your ...