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Fact Sheet
A variety of approaches target the blood stem cells to relieve or reduce your symptoms of sickle cell disease.
… cells to reduce your symptoms of sickle cell disease.  Gene therapies use your own stem cells, while bone marrow transplants use the stem cells of a matched donor. Gene therapy  In gene therapy , your stem cells are changed by …
News Release
NHGRI researchers have identified new genes associated with the Erdheim-Chester disease (ECD) and some possible new therapies.
… Institute (NHGRI) researchers have identified new genes associated with the Erdheim-Chester disease (ECD) and some … and protect the body from infection. ECD has no standard therapy, although consensus guidelines for clinical … were available for molecular testing, half had BRAF V600E gene mutations, which is sometimes seen in colon cancer, lung …
Talking Glossary
Un virus es un microorganismo infeccioso que consta de un segmento de ácido nucleico (ADN o ARN) rodeado por una cubierta proteica.
… Virus … Un virus es un microorganismo infeccioso que consta de un … (ADN o ARN) rodeado por una cubierta proteica. … A virus is an infectious microbe consisting of a segment of … acid (either DNA or RNA) surrounded by a protein coat. A virus cannot replicate alone; instead, it must infect cells …
News Release
The U.S. National Institutes of Health (NIH) has long invested in basic genetics and genomics research, clinical trials, as well as translational medicine and social science studies, to advance our understanding of this widespread illness to help develop effective therapies.
… the U.S. Food and Drug Administration (FDA) approved two gene therapies for the treatment of sickle cell disease in … The Democratizing Education for Sickle Cell Disease Gene Therapy Project , a collaborative effort that aims to help … will minimize or eliminate potential risks that might be associated with these therapies." —Gary H. Gibbons, M.D., …
Educational Resources
Gene regulation is the process of turning genes on and off.
… Gene Regulation … Gene regulation is the process of turning genes on and off. … Gene Expression, Genes, DNA, Transcription Factors … Gene regulation is the process used to control the timing, …
Educational Resources
The gene is the basic physical unit of inheritance.
… Gene … The gene is the basic physical unit of inheritance. … Inheritance, Gene, Double Helix, DNA, DNA Sequence … The gene is considered the basic unit of inheritance. Genes are …
Health FAQ
Discuss these questions with your doctor or health care provider.
… welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to … Primary care during and after the gene therapy.  Costs associated with caregivers who will provide you support (for …
Educational Resources
A candidate gene is a gene whose chromosomal location is associated with a particular disease or other phenotype.
… Candidate Gene … A candidate gene is a gene whose chromosomal location is associated with a particular disease or other phenotype. … … … A candidate gene is a gene whose chromosomal location is associated with a particular disease or other phenotype. … …
News Release
NIH and Inova Health are launching The Genomic Ascertainment Cohort, a pilot project that will examine gene and gene variant influence on phenotypes.
… and investigate the health consequences of their genes and gene variants. … Reversing a Long-Standing Paradigm To … them to recall genotyped people and examine the genes and gene variants' influence on their phenotypes, an individual's … traits or symptoms and then searching for genes or gene variants that cause or contribute to them. NIH will …
Educational Resources
Gene environment interaction is an influence on the expression of a trait that results from the interplay between genes and the environment.
… Gene Environment Interaction … Gene environment interaction is an influence on the … … Genes, Environmental Factors, Genetics, DNA … Gene–environment interaction refers to the interplay of genes … ultraviolet radiation from the sun and social context. … Gene environment interaction is an influence on the …