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UrduThe Mount Sinai Health System has received a $12,180,625 grant from the National Heart, Lung and Blood Institute to compare new treatment options for sickle cell disease and determine which work best for specific patients.
Tag: Blood Disorders
Mount Sinai Scientists Unlock Secrets of Red Blood Cell Transporter, Potentially Paving the Way for New Drugs
Researchers at the Icahn School of Medicine at Mount Sinai have identified the structure of a special transporter found in red blood cells and how it interacts with drugs.
Research sheds new light on gene therapy for blood disorders
A study shows new steps toward more patients getting gene therapy
Does That MDS Diagnosis Need a Second Opinion?
Blood disorders known as myelodysplastic syndromes/neoplasms are commonly misdiagnosed – putting patients at increased risk for treatment mistakes and other potentially harmful consequences. A new study highlights the vital need for strong coordination between clinicians and skilled pathologists to ensure accurate, timely diagnosis of blood cancers.
CHOP and Penn Medicine Researchers Develop “In Vivo” RNA-based Gene Editing Model for Blood Disorders
In a step forward in the development of genetic medicines, researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body. If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant. The findings were published today in the journal Science.
CHOP Researchers Develop Proof-of-Concept Treatment that Elevates Both Adult and Fetal Hemoglobin
Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin. Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector. The results were published in Haematologica.
Researchers Discover Common Mechanism Causing Autoimmune Disease and Blood Cancers
In a study by Yale Cancer Center, researchers report on the discovery of a common mechanism that promotes both autoimmune diseases and blood cancers, including the blood diseases Acute Lymphoblastic Leukemia (ALL), Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL).
Cecelia Calhoun Named Director of the Adolescent-Young Adult Sickle Cell Program at Yale Cancer Center and Smilow Cancer Hospital
Cecelia Calhoun, MD, MSPH, MBA, has been appointed Assistant Professor of Medicine (Hematology) and Assistant Professor of Pediatrics (Hematology/Oncology) at Yale Cancer Center and Smilow Cancer Hospital
Yale Study Shows Combined Liver–Cytokine Humanization Rescues Circulating Red Blood Cells for Testing of Novel Therapeutics
In a new study by the Yale Department of Immunobiology and Yale Cancer Center, researchers report combined liver and growth factor humanization enhances human red blood cell production and survival in circulation the immunodeficient murine host.
Researchers Discover Mechanism to Overcome Drug-Resistance in B-Cell Acute Lymphoblastic Leukemia
In a new study led by Yale Cancer Center, researchers have discovered a novel metabolic gatekeeper mechanism for leukemia. This mechanism depends on a molecule called PON2, which could lead to a new treatment for the disease. The findings were published online today in the Proceedings of the National Academy of Sciences.
Researchers discover long-sought mechanism behind most severe cases of a common blood disorder
G6PD deficiency affects about 400M people worldwide and can pose serious health risks. Uncovering the causes of the most severe cases could finally lead to treatments.
Dr. Stephanie Halene Appointed Chief of Hematology at Yale Cancer Center and Smilow Cancer Hospital
Following an extensive national search, Stephanie Halene, MD, PhD, has been appointed Chief of Hematology at Yale Cancer Center, Smilow Cancer Hospital and the Yale School of Medicine Department of Internal Medicine.
Yale Cancer Center Study Shows Caplacizumab Not Cost Effective for Rare Blood Disorder
In a new study by Yale Cancer Center researchers, the drug caplacizumab is shown not to be cost-effective in treating thrombotic thrombocytopenic purpura (TTP) when added to the standard-of-care.
UT Southwestern Leads National Efforts Around Childhood Blood Disorders
DALLAS – Oct.15, 2020 – When a child has a rare blood disorder, clinicians can struggle to find the best diagnostic and treatment methods. New research led by UT Southwestern shows the effectiveness of a treatment for aplastic anemia and reveals the range of diagnosis and treatment options used by hospitals around the country for a related disease – myelodysplastic syndrome (MDS).
Six patients with rare blood disease are doing well after gene therapy clinical trial
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.
Likelihood of Prenatal Screening for Blood Disorders Varies Between Type of Healthcare Provider
A Rutgers Cancer Institute of New Jersey investigator and other collaborators examined prenatal screening practices for blood disorders between different types of care providers and found different variations in prenatal guidance.
Peering into a more ‘human’ petri dish
The recent development of physiologic cell culture media, which models in vivo conditions, holds immense potential to improve understanding of human biology.