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Fact Sheet
Sickle cell disease resources for patients
… Education Project welcomes your feedback about the sickle cell disease gene therapy resources. Please email your … your general understanding of gene therapy for sickle cell disease. We encourage you to use these educational … What is Sickle Cell Disease?   National Institutes of Health (NIH)  About …
Research at NHGRI
The Democratizing Education for Sickle Cell Disease Gene Therapy project developed patient-focused educational materials for the sickle cell community.
… The Big Picture Sickle cell disease is an inherited disorder caused by a change in … (FDA) approved two gene therapies to treat sickle cell disease. More than 50 scientists, physicians, patients … develop content for the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in …
Talking Glossary
Chromosomes are threadlike structures made of protein and a single molecule of DNA that serve to carry the genomic information from cell to cell.
… Chromosomes are threadlike structures made of protein and a single molecule of DNA that serve to carry the genomic information from cell to cell. … Chromosomes are threadlike structures made of protein and a single molecule of DNA that serve to carry the genomic …
Fact Sheet
Gene therapy for sickle cell disease is available to you through FDA-approved therapies and through clinical trials.
… Gene therapy for sickle cell disease is available to you through FDA-approved … . While there are a number of clinical trials for sickle cell gene therapy, the journey can be similar across all … therapy may depend on: Your age. Your diagnosis of sickle cell disease. Your response to other treatments. Whether you …
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.
… People with sickle cell disease (SCD) can experience excruciating pain all over … severity of SCD symptoms. "Scientists identified sickle cell anemia in 1910, but there is still much to learn about … Normal blood cells (left) and the blood cells in Sickle cell disease, which do not flow through the circulatory …
Educational Resources
Messenger RNA (abbreviated mRNA) is a type of single-stranded RNA involved in protein synthesis.
… Messenger RNA (abbreviated mRNA) is a type of single-stranded RNA involved in protein synthesis. … Messenger RNA, MRNA, Protein Synthesis, Cell Biology … Messenger RNA (abbreviated mRNA) is a type of single-stranded RNA involved in protein synthesis. mRNA is … of mRNA is to carry protein information from the DNA in a cell’s nucleus to the cell’s cytoplasm (watery interior), …
News Release
Researchers explored patients', parents' and physicians' perspectives on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease.
… on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease (SCD). … Heart, Lung, and Blood Institute launched the Cure Sickle Cell Initiative  to accelerate development of the most … Mediterranean basin, Middle East and India, is caused by a single gene mutation that causes red blood cells to take on …
Educational Resources
Sickle cell disease is a hereditary disease seen most often among people of African ancestry.
… Sickle Cell Disease … Sickle cell disease is a hereditary disease seen most often among … Sickle cell disease is a hereditary disease seen most often among … on an unusual sickle shape. Individuals affected by sickle cell disease are chronically anemic and experience …
Research Funding
A list of members who contribute to the goals of the IGVF Consortium by generating data and/or analyses, sharing data and/or analyses freely through the IGVF Data and Administrative Coordinating Center (DACC), and/or by contributing to cross- consortium integrative analyses.
… University The Brennand laboratory integrates human stem cell models and genomic engineering, towards resolving the … Children’s Hospital Medical Center The Grimes lab applies single-cell technologies, computational genomics and systems … The McGrail lab integrates multi-omic, systems-level analyses with experimental biology in order to understand how …
News Release
NHGRI researchers have released new educational materials to help the sickle cell disease community learn about gene therapies for the disease.
… emerging developments in gene therapies for sickle cell disease. … New educational materials to help the sickle cell disease community learn about gene therapies for the … of Health. The Democratizing Education for Sickle Cell Disease Gene Therapy Project, led by Vence Bonham Jr., …