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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.
Staff
Dr. Colin Fletcher is the program director in the Knockout Mouse Project (KOMP) in NHGRI's Extramural Research Program.
… 8,500 knockouts of mouse genes, in the form of embryonic stem (ES) cells. He is now working to implement The Knockout Mouse … Mouse Phenotyping Project (KOMP2), mouse genes, embryonic stem cells … Colin Fletcher, Knockout Mouse Project (KOMP), …
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 … to be produced after birth. Hemoglobin A , also known as adult hemoglobin or HbA, is typically present in red blood … types of hemoglobin that make up red blood cells. … Blood stem cells (also called hematopoietic stem cells) are cells …
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 …
Educational Resources
The nucleolus is a region found within the cell nucleus that is concerned with producing and assembling the cell's ribosomes.
Fact Sheets
A transcriptome is a collection of all the gene readouts present in a cell.
… contains the instructions needed to build and maintain cells. These instructions are spelled out in the form of … when and where each gene is turned on or off in the cells and tissues of an organism. Depending on the technique … nearly every cell contains the same genes, but different cells show different patterns of gene expression. These …