Researchers at Harvard Medical School and Dana-Farber Cancer Institute report that humans have a surprisingly high number of genes in which one of two copies has been randomly silenced. They suggest that the phenomenon is ...
Feb 4, 2016
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In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering—by editing her genome.
Dec 5, 2019
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Therapeutic use of gene editing with the CRISPR-Cas9 technique may inadvertently increase the risk of cancer, according to a new study from Karolinska Institutet, Sweden, and the University of Helsinki, Finland, published ...
Jun 11, 2018
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A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
Mar 1, 2017
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Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide, especially Black people.
Dec 5, 2020
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A series of laboratory studies is underway in the United States to improve gene therapy worldwide for sickle cell disease, a complex and sometimes deadly heritable blood disorder that dramatically affects the structure and ...
A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of ...
Oct 12, 2016
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Asymptomatic sickle cell disease patients actually lack a tiny part of the genome, UNSW scientists have shown.
Apr 8, 2022
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Gene therapy holds a lot of promise in medicine. If we could safely alter our own DNA, we might eliminate diseases our ancestors passed down to us.
Nov 27, 2018
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Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. These diseases, which once would require a bone marrow ...
Oct 18, 2019
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