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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
A mutation is a change in a DNA sequence. Mutations can result from DNA copying mistakes made during cell division, exposure to ionizing radiation, exposure to chemicals called mutagens, or infection by viruses.
… or infection by viruses. … Mutation, DNA Sequence, Germ Line, Gene … A mutation is a change in the DNA sequence … to offspring, while somatic mutations (that occur in body cells) are not passed on. … A mutation is a change in a DNA …
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 blood cells need hemoglobin to deliver oxygen to your body’s organs … known as fetal hemoglobin or HbF, is present in red blood cells before birth (during fetal development). After birth, a …
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 …
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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
Transgenic means that one or more DNA sequences from another species have been introduced by artificial means.
… means. … Cell, Cloning, Deoxyribonucleic Acid (DNA), Gene, Germ Line … Transgenic refers to an organism or cell whose …
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.