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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.
… Cell-Free DNA Testing … Cell-free DNA testing is a laboratory method that involves … associated with a hereditary or genetic disorder. … Cell-free DNA testing is a laboratory method that involves … a hereditary or genetic disorder. For example, prenatal cell-free DNA testing is a non-invasive method used during …
Fact Sheet
Today, gene therapy for sickle cell disease is considered experimental and only available to you if you participate in a clinical trial.
… may be dependent on: Your age. Your diagnosis of sickle cell disease. Your response to other treatments. Whether you … a clinical trial.  …  Step 4: Preparation for stem cell collection (~90 days)  Gene therapy requires stem cells … of sickled blood cells.  …  … ​​​​​ … Step 5: Stem cell collection (~6–8 weeks)  The stem cell collection …
Educational Resources
Messenger RNA (abbreviated mRNA) is a type of single-stranded RNA involved in protein synthesis.
… RNA (mRNA) … 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 …
News Release
Genomic analyses turned up genes that belong to inflammatory pathways.
… translated). In this case, the effect leads to a form of cell death that causes inflammation. Dr. Kastner had a hunch … Center staff. "The genes that turned up in the genomic analyses of the disease we focus on belong to inflammatory … odyssey ends at the NIH Clinical Center … Genomic analyses turned up genes that belong to inflammatory …
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). … A First-of-its-Kind Study CRISPR-Cas9 … 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
Ribonucleic acid (RNA) is a molecule similar to DNA. Unlike DNA, RNA is single-stranded.
… (RNA) is a molecule similar to DNA. Unlike DNA, RNA is single-stranded. … Cell, Genetic Code, Deoxyribonucleic Acid (DNA), Protein, … to DNA. Unlike DNA, however, RNA is most often single-stranded. An RNA molecule has a backbone made of …
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., …
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 … Gene, Inherited, Mutation, Protein, Recessive … Sickle cell disease is a hereditary disease seen most often among …
Fact Sheet
Currently, there are different gene therapy approaches available through clinical trials.
… Hemoglobin types and sickle cell disease  Your red blood cells contain hemoglobin , a … oxygen to your body’s organs and tissues. Every red blood cell contains thousands of hemoglobin molecules.  The genes … to instead make hemoglobin A. For individuals with sickle cell disease, a small amount of HbF continues to be produced …
Educational Resources
Apoptosis is the process of programmed cell death.
… Apoptosis … Apoptosis is the process of programmed cell death. … Programmed Cell Death, Development, DNA Repair, Cancer, Tumor, Cell Division … Apoptosis is the process of programmed cell …