Sentences with word «thalassemia»

I've probably had over 10,000 needle sticks throughout my lifetime, because I have a genetic illness called Beta Thalassemia Major.

He explains that he «was attracted by the idea of using mouse models to answer human questions» and «could also see the possibility of applying the results in the form of gene therapy» for thalassemia patients.

The aim of present study was to evaluate the association of thalassemia minor with suicide, impulsivity and ag - gression.

If the cells are small, suggesting b thalassemia minor, further testing can be done to confirm the diagnosis.

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.

A person with b thalassemia major, in which both genes are affected, develops severe anemia starting at a few months of age.

People who have an absence or severe abnormality of only one of their b globin genes have b thalassemia minor.

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

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.

Since antique DNA (aDNA) of malaria is very difficult to extract, they studied thalassemia and other genetic adaptations in its place.

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

Dr. Sadelain is the recipient of the Cancer Research Institute's William B. Coley Award for Distinguished Research in Tumor Immunology, the Sultan Bin Khalifa International Award for Innovative Medical Research on Thalassemia, and the New York Intellectual Property Law Association Inventor of the Year Award.

Abbott chairs the All - Party Parliamentary British - Caribbean Group and the All - Party Sickle Cell and Thalassemia Group.

Correction of β - thalassemia mutant by base editor in human embryos.

Even today, such thalassemias occur relatively frequently in former malaria regions, such as the Mediterranean.

Prenatal cell therapy failed for thalassemia nearly 30 years ago, but Mackenzie hopes for a different outcome this time.

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.

Found in patients predominantly of Mediterranean, African, Middle Eastern and Asian descent, β thalassemia affects thousands of infants worldwide each year, according to the NHLBI.

Stem cells may be used in conjunction with gene therapy to correct inherited diseases such as thalassemia [a blood disease that causes anemia]-- it would be wonderful to be able to apply a single treatment fairly early on in a child's life and have it be fully effective from then on.

Thalassemias differ in severity and in the type of globin chain that is deficient.

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.

Could Anti-Erythropoietin Receptor Antibodies Be the Future Management of Beta Thalassemia Intermedia?

Successful treatment of murine ß - thalassemia using in vivo selection of genetically - modified, drug resistant hematopoietic cells.

The degree of phenotypic correction of murine ß — thalassemia intermedia following lentiviral - mediated transfer of a human γ — globin gene is influenced by chromosomal position effects and vector copy number.

Seamless gene correction of β - thalassemia mutations in patient - specific iPSCs using CRISPR / Cas9 and piggyBac.

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.

Biotech CRISPR Therapeutics is also joining the queue, as they use a similar approach in a clinical trial for beta thalassemia that's slated to begin this year in Europe.

These samples, known as Guthrie cards, or blood spots, are routinely drawn from newborns, with a prick to the heel, in order to diagnose such genetic disorders as thalassemia and Phenylketonuria, or PKU.

One of the most common causes thalassemia, a type of anemia that occurs more often in parts of Africa, the Mediterranean, the Middle East, and Southeast Asia than it does elsewhere.

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

Patients with b thalassemia major can survive only with the help of frequent blood transfusions.

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.

«Results presented by [CRISPR Therapeutics] at a haematology meeting showed that the method dramatically increased foetal haemoglobin in β - Thalassemia patients» cells.

If the strategy proves effective in clinical studies, it could lead to the development of gene therapy for people with thalassemia, and potentially sickle cell disease and other inherited blood disorders, he said.

In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta - thalassemia, and myelofibrosis.

Interim data for LentiGlobin to eliminate or reduce chronic blood transfusions in patients with transfusion - dependent beta - thalassemia was also published in the New England Journal of Medicine.

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.

Bluebird Bio (BLUE)- The company's data in beta - thalassemia was highlighted in the Wall Street Journal.

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.

In many states, the test is required and is frequently done in conjunction with several other tests, such as Galactosemia, Thalassemia, etc..

Chiara was never an appropriate candidate for homebirth because of a pre-existing medical condition (beta - thalassemia), but:

For example, women of African or Mediterranean descent are usually tested for thalassemia or sickle cell trait or disease because they're at higher risk of these chronic blood diseases or carrying the sickle cell trait, which can be passed on to their children.

Then a team of Chinese researchers used that base editor to correct a mutation in human embryos that causes the blood disorder beta - thalassemia, reported September 23 in Protein & Cell (SN: 11/25/17, p. 7).

When that gene is mutated, it causes an inherited blood disorder called beta thalassemia.

The researchers say the study represents a proof of concept for the repair of blood - borne genetic diseases, such as sickle cell disease and thalassemia.

- thalassemia, which is dominant on Sardinia.