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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., …
Fact Sheet
A variety of approaches target the blood stem cells to relieve or reduce your symptoms of sickle cell disease.
… It is important to understand the components of gene therapy, how these components differ and other treatment … cells to reduce your symptoms of sickle cell disease.  Gene therapies use your own stem cells, while bone marrow …
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, …
Fact Sheet
Your mental and physical health are deeply connected. Mental health is important for your overall health and quality of life.
… actions you may want to take as you think about receiving gene therapy. …  There are many unknowns about what to expect from gene therapy. Your thoughts, feelings, beliefs and attitudes …
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 …
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. … …
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 …
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 …