One study was done on kids
with Thalassemia, a genetic type of anemia, who need frequent blood transfusions.
One study was done on children
with Thalassemia, a genetic form of anemia, who require regular blood transfusions.
To achieve gene editing in mice
with thalassemia, professor of therapeutic radiology and of genetics Peter M. Glazer, M.D. and his co-authors developed an alternative approach using a novel combination of nanoparticles, synthetic pieces of DNA, and a simple IV injection.
Not exact matches
In many states, the test is required and is frequently done in conjunction
with several other tests, such as Galactosemia,
Thalassemia, etc..
Harvard Stem Cell Institute (HSCI) scientists have taken the first steps toward developing a treatment that would make bone marrow — blood stem cell — transplantation safer and, as a result, more widely available to the millions of people living
with blood disorders like sickle cell anemia,
thalassemia, and AIDS.
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.
The team first created embryos
with genetic mutations that caused two different diseases: β -
thalassemia and favism (an anemia caused by eating fava beans).
In contrast to Ciclopirox, approved for topical use, Deferiprone is FDA - and EMA - approved for systemic use (in certain
thalassemia patients
with iron overload).
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.
A person
with b
thalassemia major, in which both genes are affected, develops severe anemia starting at a few months of age.
Another danger for people
with b
thalassemia is an iron overload from the gradual breakdown of the transfused blood.
Patients
with b
thalassemia major can survive only
with the help of frequent blood transfusions.
«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 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.
Currently
with St. Jude Children's Research Hospital, Cunningham has led research that expands medical understanding of sickle cell anemia and
thalassemia.
The BBC reports that gene therapy has been used successfully to treat a patient
with severe β -
thalassemia.
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.