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.
Brazilian researchers propose new way for treating thalassemia major

Brazilian researchers propose new way for treating thalassemia major

Epigem challenges Durham University students to develop diagnostic devices for rare anaemic conditions

Epigem challenges Durham University students to develop diagnostic devices for rare anaemic conditions

Yale researchers use new gene editing technique to correct mutations that cause thalassemia

Yale researchers use new gene editing technique to correct mutations that cause thalassemia

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

Scientists find new way to use CRISPR gene editing to help fix sickle cell disease

Scientists find new way to use CRISPR gene editing to help fix sickle cell disease

New computer model shows how spleen filters misshapen red blood cells

New computer model shows how spleen filters misshapen red blood cells

Scientists develop non-toxic transplantation procedure using antibodies to target blood stem cells in mice

Scientists develop non-toxic transplantation procedure using antibodies to target blood stem cells in mice

Minihepcidins may potentially treat serious genetic blood diseases in children, adults

Minihepcidins may potentially treat serious genetic blood diseases in children, adults

Key opinions leaders to discuss role of cyclophosphamide in the field of HSCT

Key opinions leaders to discuss role of cyclophosphamide in the field of HSCT

Sangamo presents immunological data from SB-728-T HIV clinical study at CROI 2016

Sangamo presents immunological data from SB-728-T HIV clinical study at CROI 2016

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

New method may extend use of noninvasive prenatal testing to detect chromosomal abnormalities

New method may extend use of noninvasive prenatal testing to detect chromosomal abnormalities

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Americord Registry, Smart Cells International partner to educate parents on benefits of cord blood banking

Americord Registry, Smart Cells International partner to educate parents on benefits of cord blood banking

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment