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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 …
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 …
Educational Resources
Mitochondria are membrane-bound cell organelles (mitochondrion, singular) that generate most of the chemical energy needed to power the cell's biochemical reactions.
Educational Resources
A centrosome is a cellular structure involved in the process of cell division.
… help separate the replicated chromosomes into the daughter cells. … A centrosome is a cellular structure involved in the …