Sentences with phrase «thalassemia patients»
«Results presented by [CRISPR Therapeutics] at a haematology meeting showed that the method dramatically increased foetal haemoglobin in β - Thalassemia patients» cells.
http://www.frontlinegenomics.com/
In contrast to Ciclopirox, approved for topical use, Deferiprone is FDA - and EMA - approved for systemic use (in certain thalassemia patients with iron overload).
Not exact matches
Patients with b thalassemia major can survive only with the help of frequent blood transfusions.
Other blood disorders that have shown significant benefit from targeted gene therapy in small trials include hemophilia (specifically, factor IX deficiency), severe beta - thalassemia (deficiency for the adult beta - globin gene) and leukemia, where the patient's immune cells were treated to enable them to recognize cancer cells and destroy them.
-- The NEJM published updates from two early - stage Bluebird Bio (NASDAQ: BLUE) studies of a gene therapy for the rare disease beta - thalassemia, which requires patients to... Next Page»
«For example, despite matching the ABO blood group antigens during red blood cell (RBC) transfusion, up to 45 percent of chronically transfused patients, such as those with sickle cell disease or thalassemia, develop antibodies to mismatched minor antigens on transfused RBCs; this process is known as alloimmunization.»
The Bone Marrow and Stem Cell Transplant Program is expanding the use of this technique for patients with solid tumors including neuroblastoma and brain tumors; a variety of high - risk hematologic diseases, such as thalassemia major and transfusion - dependent sickle cell disease; and other nonmalignant diseases.
The BBC reports that gene therapy has been used successfully to treat a patient with severe β - thalassemia.
Seamless gene correction of β - thalassemia mutations in patient - specific iPSCs using CRISPR / Cas9 and piggyBac.
Beta - Globin Haplotypes and Alpha - Thalassemia 3.7 kb Deletion in Sickle Cell Disease Patients From the Occidental Brazilian Amazon
We describe βS and βC haplotypes and α - thalassemia 3.7 kb genotypes from SCD patients Fundacao Hospitalar de Hematologia e Hemoterapia do Amazonas, Manaus, AM.
Less than 2 % of diseases, such as cystic fibrosis, Huntington's chorea, and beta thalassemia, result from a single faulty gene, and only about 5 % of cancer patients can attribute their diseases to heredity.