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Research at NHGRI
The Democratizing Education for Sickle Cell Disease Gene Therapy project utilizes a deliberative stakeholder-engagement approach to develop patient educational materials for sickle cell disease gene therapy.
… to five educational focus areas: Understanding the red blood cell Understanding gene therapy approaches Clinical …
News Release
NHGRI researchers seek help from people with Sickle Cell Disease to find the factors - environmental, social and genetic - that impact the severity of symptoms.
… ulcers. These and other symptoms result when mutated red blood cells take a sickle shape, blocking blood flow and decreasing oxygen delivery. Little is known …
Genetic Disorders
Thalassemia is a group of inherited diseases of the blood that affect a person's ability to produce hemoglobin, resulting in anemia.
… Thalassemia is a group of inherited diseases of the blood that affect a person's ability to produce hemoglobin, … is actually a group of inherited diseases of the blood that affect a person's ability to produce hemoglobin, … resulting in anemia. Hemoglobin is a protein in red blood cells that carries oxygen and nutrients to cells in the body. …
News Release
A simple blood draw is replacing a giant needle to the belly for testing their fetuses for a range of genetic disorders.
… breathing a collective sigh of relief now that a simple blood draw is replacing a giant needle to the belly for … for a range of genetic disorders. This non-invasive blood test, combined with advances in genomic medicine, has … they view the related ethical issues." Through a simple blood test, physicians will soon be able to determine the …
Educational Resources
An antibody is a protein component of the immune system that circulates in the blood, recognizes foreign substances like bacteria and viruses, and neutralizes them.
… component of the immune system that circulates in the blood, recognizes foreign substances like bacteria and … and neutralizes them. … Immune System, Bacteria, Virus, Blood, Circulation, Disease, Antigen … An antibody is a … component of the immune system that circulates in the blood, recognizes foreign substances like bacteria and …
Talking Glossary
Cell-free DNA testing is a laboratory method that involves analyzing free (i.e., non-cellular) DNA contained within a biological sample, most often to look for genomic variants associated with a hereditary or genetic disorder.
Fact Sheet
Today, gene therapy for sickle cell disease is considered experimental and only available to you if you participate in a clinical trial.
… also be required to have: Certain screenings (e.g., MRI, blood tests). A genetic test to determine if there are … cell collection (~90 days) Gene therapy requires stem cells to be collected from your bone marrow or blood, taken to a lab for modification, then returned to you. …
Staff
Dr. David Bodine is the chief of and senior investigator in NHGRI's Genetics and Molecular Biology Branch.
… After postdoctoral work at NIH's National Heart, Lung and Blood Institute, Dr. Bodine founded the Hematopoiesis Section … College in 2013 for his contributions to the genetics of blood disorders. As a graduate student, he was the recipient … process of generating all of the different types of blood cells (erythrocytes, lymphocytes, platelets, etc.) …
Fact Sheet
A variety of approaches target the blood stem cells to relieve or reduce your symptoms of sickle cell disease.
… Types of treatments A variety of approaches target the blood stem cells to reduce your symptoms of sickle cell disease. Gene … aim to decrease the amount of hemoglobin S in your red blood cells. If they successfully decrease the hemoglobin S, …
News Release
NHGRI researchers have released new educational materials to help the sickle cell disease community learn about gene therapies for the disease.
… of Health, particularly the National Heart, Lung, and Blood Institute.” “Most importantly, the project included … of the disease comes from the “sickle” shape of the red blood cells that form in patients with the disorder. Red blood …