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Fact Sheet
Today, gene therapy for sickle cell disease is considered experimental and only available to you if you participate in a clinical trial.
… leaving) a clinical trial.   …   Step 4: Preparation for stem cell collection (~90 days)   Gene therapy requires stem cells to be collected from your bone marrow or blood, taken …
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.
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 protein that carries oxygen. Red … types of hemoglobin that make up red blood cells. … Blood stem cells (also called hematopoietic stem cells) are cells that can develop into any type of blood …
News Release
NHGRI researchers have released new educational materials to help the sickle cell disease community learn about gene therapies for the disease.
… the disease comes from the “sickle” shape of the red blood cells that form in patients with the disorder. Red blood cells contain the protein hemoglobin, which carries oxygen in …
News Release
Researchers explored patients', parents' and physicians' perspectives on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease.
… is caused by a single gene mutation that causes red blood cells to take on an unusual sickle shape. People affected by …
Educational Resources
Sickle cell disease is a hereditary disease seen most often among people of African ancestry.
… recessive trait. The mutation causes the red blood cells to take on an unusual sickle shape. Individuals …
Educational Resources
Apoptosis is the process of programmed cell death.
… It is used during early development to eliminate unwanted cells; for example, those between the fingers of a developing … hand. In adults, apoptosis is used to rid the body of cells that have been damaged beyond repair. Apoptosis also …
Clinical Research
The INSIGHTS study is exploring sickle cell disease in adults.
Educational Resources
The nucleolus is a region found within the cell nucleus that is concerned with producing and assembling the cell's ribosomes.
Staff
Dr. Paul Liu is a senior investigator in NHGRI's Translational and Functional Genomics Branch. ​
… during leukemia development by microarray analysis with cells from Cbfb-MYH11 knock-in mice, and identified … mouse models. The zebrafish is a useful model for studying embryonic development and is easily amenable to large-scale … made novel findings regarding its role in hematopoietic stem cell production (Bresciani, Blood in press). Overall the …