Thalassemia News and Research

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Thalassemia is a group of genetic blood disorders that affect approximately 1,000 individuals in the United States. The most severe of these disorders is Cooley's Anemia.

People with thalassemia have a genetic defect of their red blood cells that affects the cells' ability to produce normal hemoglobin. Red blood cells use hemoglobin to carry oxygen to tissues. As a result of the defect, most forms of thalassemia produce a chronic, lifelong anemia that begins in early childhood and often must be treated with frequent transfusions.
Mystery surrounds gene edited babies one year on

Mystery surrounds gene edited babies one year on

Pioneering CRISPR therapy leaves two patients free of blood disease symptoms

Pioneering CRISPR therapy leaves two patients free of blood disease symptoms

Deterministic mechanoporation of cells en masse to cure cancer

Deterministic mechanoporation of cells en masse to cure cancer

Carnegie Mellon and Yale win NIH grant to advance gene editing technique

Carnegie Mellon and Yale win NIH grant to advance gene editing technique

Researchers make breakthrough in gene therapy for sickle cell disease

Researchers make breakthrough in gene therapy for sickle cell disease

Genetic engineering could open possibilities for patients with Parkinson’s disease

Genetic engineering could open possibilities for patients with Parkinson’s disease

First ever American gene-editing treatment using CRISPR for genetic disease

First ever American gene-editing treatment using CRISPR for genetic disease

Better education and communication critical for increasing blood donation among minorities

Better education and communication critical for increasing blood donation among minorities

More screening and new treatments needed for sickle cell disease

More screening and new treatments needed for sickle cell disease

Scientists use gold nanoparticles to edit genes tied to HIV, inherited blood disorders

Scientists use gold nanoparticles to edit genes tied to HIV, inherited blood disorders

Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia

Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia

New gene therapy cures babies with fatal 'Bubble Boy' disease

New gene therapy cures babies with fatal 'Bubble Boy' disease

Researchers develop new strategy to treat sickle cell disease and beta thalassemia

Researchers develop new strategy to treat sickle cell disease and beta thalassemia

Study illuminates how hot spots of genetic variation evolved in the human genome

Study illuminates how hot spots of genetic variation evolved in the human genome

Deadly inherited blood disorders cured with bone marrow transplant

Deadly inherited blood disorders cured with bone marrow transplant

Antibody-based treatment could improve cure rate for blood and immune disorders

Antibody-based treatment could improve cure rate for blood and immune disorders

App that could detect level of anemia by looking at fingernails

App that could detect level of anemia by looking at fingernails

Advances in gene therapy for inherited blood disorders

Advances in gene therapy for inherited blood disorders

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy