Sentences with phrase «disease thalassemia»

Professor Capechi has shed light on the causes of several human birth abnormalities, while Professor Smithies has worked on the blood disease thalassemia and hypertension.

Not exact matches

bluebird bio (BLUE)- The company's BCMA CAR - T drug candidate bb2121 remains attractive despite overblown fears on durability (peak sales of $ 2 billion or more), LentiGlobin has a good shot at success in TDT (Transfusion - Dependent ß - Thalassemia) and SCD (Severe Sickle Cell Disease), and they have a strong cash position.

Last month she filed a request with the U.S. Food and Drug Administration to offer hematopoietic stem cells to fetuses with α thalassemia, a severe form of the disease that is fatal before or soon after birth.

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.

The disease β - thalassemia, a blood disorder, is caused by a mistake in a single DNA base.

Many of them were babies, and a few suffered from the hematologic disorders that have been the focus of Daley's research — diseases like Fanconi's anemia, thalassemia, sickle - cell anemia, and childhood leukemias.

The team first created embryos with genetic mutations that caused two different diseases: β - thalassemia and favism (an anemia caused by eating fava beans).

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.

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.

-- 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).

In a recent study, Chinese researchers used CRISPR / Cas in human embryos to correct a mutation that causes the blood disease beta - thalassemia.

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).

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.

Beta - Globin Haplotypes and Alpha - Thalassemia 3.7 kb Deletion in Sickle Cell Disease Patients From the Occidental Brazilian Amazon

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.

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.

Anemia can be triggered by blood loss, a folic acid or vitamin B12 deficiency, sickle cell disease, and a genetic disorder called thalassemia, among other causes.

Some of the most common Genetic Disorders include Down syndrome, Hunington's disease, Hemophilia, Thalassemia, Cystic Firbrosis and Sickle Cell Anaemia, among many others.

These diseases include neurological diseases with a disability of 40 percent or more, AIDS, Parkinson's disease, malignant cancers, Hemophilia, chronic renal failure and Thalassemia, to name a few.

Exceptionally well - versed in genetics and hematology, with great focus on determining blood diseases such as Thalassemia and Leukemia.