Sentences with phrase «for thalassemia»

Dr. Scriver also helped develop pediatric genetic screening programs, including one for thalassemia and another for Tay - Sachs disease, both targeted at high school students, which were shown to have lasting effects on personal and public health in Quebec.

Development of gene therapy for thalassemia.

Because changes in the b globin gene are common in the Mediterranean area, people whose ancestors came from such areas as Greece or Italy may wish to be screened for thalassemia.

Given new information about «The cure for thalassemia», a formal system would detect an inconsistency because the word «cure» is associated with diseases and not places.

Cord blood transplants are also accepted as treatment for thalassemia and sickle cell anemia, inherited blood disorders that are prevalent in certain ethnic groups.

Not exact matches

For instance, a couple who each suffers from beta thalassemia might only have healthy children free from the inherited blood disorder if they were able to produce embryos in which the genetic defect was corrected using gene editing.

Notably, research groups might be able to apply the approach described in this study to develop treatments for other blood diseases such as β - thalassemia, severe combined immunodeficiency (SCID), chronic granulomatous disease, rare disorders like Wiskott - Aldrich syndrome and Fanconi anemia, and even HIV infection.

But Knutson does see this finding as more relevant for possibly treating other incurable iron overload disorders such as thalassemia.

When they tried correcting the mutations using CRISPR - Cas9, their success rate was one in four for β - thalassemia and two in two for favism, they report this month in Molecular Genetics and Genomics.

In contrast to Ciclopirox, approved for topical use, Deferiprone is FDA - and EMA - approved for systemic use (in certain thalassemia patients with iron overload).

However a human could rationalise the faulty belief by reinterpreting the metaphor in a newspaper style as «the cure for the political turmoil that beset the inhabitants of thalassemia».

Boston Children's Hospital has offered non-exclusive licenses to for - profit entities on a patent developed by Orkin's laboratory regarding BCL11A, a genetic switch regulating hemoglobin production that is expected to form the basis of clinical trials for gene therapy and gene editing for sickle cell disease and thalassemia.

Another danger for people with b thalassemia is an iron overload from the gradual breakdown of the transfused blood.

For unknown reasons, the ATR - X mutation causes mild a thalassemia even though the a globin gene is normal.

Also, prenatal diagnosis is available for pregnancies at risk for severe thalassemia.

Even so, it is important to diagnose b thalassemia minor (also called thalassemia trait) because iron treatment for affected individuals does not help their anemia and may even be harmful.

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.

Deferiprone, available for the past 20 years, is used in more than 60 countries to prevent death from blood transfusion - related complications during treatment of thalassemia, an inherited blood disorder in which the body makes an abnormal form of hemoglobin.

-- 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.»

CRISPR / Cas9 could be used to develop therapies for humans for genetic blood diseases such as sickle cell or thalassemia, and this paper does not change that potential.

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 hypothesis is eminently testable as iron reduction therapy is useful for chronic diseases associated with iron excess such as nonalcoholic steatohepatitis (NASH), atherosclerosis, hereditary hemochromatosis and thalassemia.

She is registred to the National Order of Biologists in the province of Palermo; collaboration in research project from 2012 to 2015 at the Department of Biopathology and Biotechnology, University of Palermo, focusing the study on the identification of molecules capable to modulate intracellular metabolic pathways for the prevention and treatment of infectious, tumor and degenerative disease, in collaboration with Prof. Angela Santoni, University of Rome; collaboration in research project in 2011 at the hospital «Villa Sofia Cervello» of Palermo to study methods can cure the genetic defect that causes thalassemia through genetic engineering; she studies different mechanisms of the differentiation and the activation of human gammadelta T cells as effector cells of the immune response against cancer and infectious diseases; she investigates about the identification and development of biomarkers of resistance and susceptibility to Mycobacterium tuberculosis infection; Valentina Orlando has published 13 papers in peer reviewed journals and 3 comunications at national and international congress.

Gene editing has emerged as a promising strategy to treat diseases like β - thalassemia and sickle cell disease which are both caused by mutations in the gene for β - globin (HBB).

Awarded the Royal Medal by the Royal Society for his work on thalassemia; awarded the Fothergillian Prize by the London Medical Society; the Manson Medal by the Royal Society of Tropical Medicine and Hygiene and the Lasker Award.

Clinical research activities are focused on the implementation of experimental protocols based on gene or cell therapy for the treatment of genetic blood disorders such as ADA - SCID, Wiskott - Aldrich Syndrome, Metachromatic Leukodystrophy, Beta - thalassemia, Chronic granulomatous disease, Mucopolysaccharidosis Type I (Hurler).

A third trial, for beta ‐ thalassemia, started in 2015 and is showing promising preliminary results.

An authority on the study and treatment of childhood cancers, as well as the biology and therapy of hemoglobinopathies — blood diseases such as sickle cell anemia and thalassemia — Cunningham is known for his work on understanding the molecular mechanism underpinning red blood cell production.

Carrier screening for beta thalassemia and related hemoglobinopathies is recommended by ACOG via complete blood count and hemoglobin electrophoresis for people of African, Southeast Asian, Mediterranean, Middle Eastern, and West Indian descent considering having children.