Exceptionally well - versed in genetics and hematology, with great focus on determining blood diseases such
as Thalassemia and Leukemia.
Mia's statement above reminded me of an inherited trait I have which is Mediterranean anemia: Better known today
as thalassemia (or as beta thalassemia or thalassemia major).
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.
Gene editing techniques have the potential to treat blood disorders that run in families, such
as thalassemia and sickle cell anemia, but their application has been largely limited to cells in a laboratory and not living animals.
But Knutson does see this finding as more relevant for possibly treating other incurable iron overload disorders such
as thalassemia.
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..
Cord blood transplants are also accepted
as treatment for
thalassemia and sickle cell anemia, inherited blood disorders that are prevalent in certain ethnic groups.
Even today, such
thalassemias occur relatively frequently in former malaria regions, such
as the Mediterranean.
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.
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.
Other plans include using CRISPR to reverse blood disorders, such
as sickle cell anemia and beta
thalassemia, caused by mutations in the hemoglobin gene.
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».
The most common type in the United States is b
thalassemia, also known
as Mediterranean anemia in its mild form or Cooley's anemia in its severe form.
This causes only mild
thalassemia, but because other genes are deleted
as well, there are additional problems — such
as birth defects and mental retardation — that do not occur in most
thalassemias.
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.
«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 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).
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).
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.
Beta
thalassemia is a genetic disorder characterized by anemia and fatigue
as well
as bone deformities and organ problems.
However, keep ALL iron supplements away from children, even carbonyl iron, and do not take any kind of iron supplement if you have hemochromatosis, hemosiderosis or hemolytic anemia such
as sickle cell anemia or
thalassemia (aka Mediterranean anemia, a type of genetic anemia where hemoglobin is not well formed)
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.