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.
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

Therapy with L-Glutamine reduces pain in patients with sickle cell disease

Therapy with L-Glutamine reduces pain in patients with sickle cell disease

Researchers identify enzyme as potential new drug target for blood disorders

Researchers identify enzyme as potential new drug target for blood disorders

Mice study shows promise for treating genetic conditions during fetal development

Mice study shows promise for treating genetic conditions during fetal development

Research project offers hope for pediatric patients with thalassemia

Research project offers hope for pediatric patients with thalassemia

Celgene to share new and updated data around novel hematological therapies

Celgene to share new and updated data around novel hematological therapies

Smart Cells launches social campaign to support Rare Disease Day 2018

Smart Cells launches social campaign to support Rare Disease Day 2018

Company-sponsored CRISPR clinical studies to begin in 2018

Company-sponsored CRISPR clinical studies to begin in 2018

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

“Chemical surgery” on embryos to free them of a gene causing thalassemia

“Chemical surgery” on embryos to free them of a gene causing thalassemia

Metabolism can impact host's susceptibility to develop malaria

Metabolism can impact host's susceptibility to develop malaria

Leeds Teaching Hospitals to introduce private stem cell collection and storage option for patients

Leeds Teaching Hospitals to introduce private stem cell collection and storage option for patients

Study provides insight into development of red blood cells

Study provides insight into development of red blood cells

Malaria already widespread on Sardinia in the Roman period, research shows

Malaria already widespread on Sardinia in the Roman period, research shows

US scientists make advances in modifying human embryos

US scientists make advances in modifying human embryos

Infographic displays facts and information about stem cells, cord blood

Infographic displays facts and information about stem cells, cord blood

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Sangamo Therapeutics and Pfizer team up for development of Hemophilia A gene therapy

Researchers develop robust technique for efficient manufacture of red blood cells

Researchers develop robust technique for efficient manufacture of red blood cells

Innovative gene therapy allows complete remission of clinical signs of sickle cell disease

Innovative gene therapy allows complete remission of clinical signs of sickle cell disease