Grants and Grant Recipients

Grant generously funded by the family and friends of Hadi Abdur Rasheed »»» Aplastic anemia Aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (AA) is a type of bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of…

Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) results from a mutation mutation: Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation…

Aplastic anemia Aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… and myelodysplastic syndrome are diseases that affect the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. stem cells stem cells: Cells in the body that develop into other…

Immune aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (IAA) is a condition where the bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. doesn’t make enough blood cells, which can lead to low levels of red blood cells, white blood…

Autoimmunity and immunodeficiency can coexist. Inborn errors of immunity are a heterogeneous group of genetic disorders in which the immune system is disturbed, not only affecting its ability to combat pathogens, but also to trigger autoimmunity. Despite early-onset and severe disease being expected for these disorders with the advent of modern DNA sequencing technologies, an increasing number of adult-onset errors of immunity has been identified. With this proposal, we aim to assess the presence of inherited gene variants predisposing to immunodeficiency as determinants of PNH and AA. First…

Acquired aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (AA), the prototypical bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. (BM) failure syndrome, is caused by immune-mediated destruction of hematopoietic stem/progenitor cells…

This research project aims to understand the biology and genetics of the disease myelodysplastic syndrome (MDS) in young patients. MDS is normally a disease of older persons (70+), and this study aims to determine the genetics and biology of MDS in patients diagnosed between the ages of 18 and 40 years old. MDS in young people is a distinct population with a lot of different genetic problems causing illness, most being related to the cell's repair of DNA and parts of the chromosomes chromosomes: The part of the cell that contains our DNA or genetic code. . Autoimmune and…

Acquired bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… syndromes, such as myelodysplastic syndrome (MDS), are frequently caused by genetic mutations. Both the underlying mechanism and prognosis of MDS have been attributed to specific driver gene mutations, and genetic classification systems have…

Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) is a rare blood disease in which blood cells are destroyed, leading to anemia anemia: (uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low…

We discovered a way to express a gene or protein of interest in cancer cells, but not healthy normal cells. Specifically, we created synthetic (not occurring in nature) introns that we can introduce into any gene of interest, such as a “killer gene,” such that the encoded protein is produced in cancer cells carrying a defined, cancer-causing mutation mutation: Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem cell in the bone marrow…