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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 … Un virus es un microorganismo infeccioso que consta de un … nucleico (ADN o ARN) rodeado por una cubierta proteica. Un virus no puede replicarse solo; por el contrario, debe … para fabricar copias de sà mismo. Con frecuencia, un virus termina matando la célula huésped en el proceso, lo que …
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 … "The discovery of new genes associated with ECD provides hope for improving the diagnoses … were available for molecular testing, half had BRAF V600E gene mutations, which is sometimes seen in colon cancer, lung …
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 … transplants use the stem cells of a matched donor. Gene therapy  In gene therapy , your stem cells are changed by …
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 … One of the new sickle cell treatments uses the CRISPR gene-editing system, a first for humans in the U.S.  NIH … 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, … regulatory proteins and chemical modification of DNA. Gene regulation is key to the ability of an organism to …
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 can … Consider these other factors. Risks of gene therapy Check out the sickle cell disease gene therapy …
Clinical Research
MINI Section invites caregivers of children with mitochondrial disease to participate in a survey about the use of immune therapies in children with mitochondrial disease.
… Use of Immunoglobulin Therapy in the Mitochondrial Disease Community …
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 … The gene is the basic physical unit of inheritance. …
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 … The term candidate gene refers to a gene that is believed to be related to a particular trait, … 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 … They are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use … Eligibility criteria may be different for each available gene therapies, including clinical trials. However, there are …