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Fact Sheet

Navigating sickle cell disease gene therapy

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

New NIH studies seek adults and families affected by sickle cell disease/trait

NHGRI researchers seek help from people with Sickle Cell Disease to find the factors - environmental, social and genetic - that impact the severity of symptoms.

… Jeannine Mjoseth … 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 …

Talking Glossary

Cell-Free DNA Testing

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 …

Educational Resources

Messenger RNA (mRNA)

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

Sickle cell disease community hopeful genome editing will rechart course of disease

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)

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 project creates new educational materials for sickle cell disease community

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., …

News Release

One patient's diagnostic odyssey ends at the NIH Clinical Center

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

Genetic carriers for sickle cell disease have higher risks of blood clots across diverse ancestries

NIH researchers and collaborators have found that that sickle cell trait is prevalent among diverse human populations.

… Study finds that sickle cell trait is prevalent among diverse human populations. … … collaborators have found that being a carrier for sickle cell disease, known as having sickle cell trait , increases … also known as venous thromboembolism. Through statistical analyses, participants were grouped based on their genetic …

Educational Resources

Sickle Cell Disease

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 …