A new study by scientists at UMass Medical School shows that using a technique called "nuclease-free" gene editing to correct cells with the mutation that causes a rare liver disease leads to repopulation of the diseased ...
Oct 20, 2017
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A Yale-led team of researchers has identified specific gene combinations that can cause deadly brain cancer glioblastoma, using new technology that can also pinpoint triggers of other types cancers, they report Aug. 14 in ...
Aug 14, 2017
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Gene editing is one of the scarier things in the science news, but not all gene editing is the same. It matters whether researchers edit "somatic" cells or "germline" cells.
Mar 18, 2019
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After a progressive weakening of the muscles takes away their motor skills, and then their abilities to stand and walk, most males with Duchenne muscular dystrophy die of heart and respiratory failure in their 20s.
Apr 6, 2018
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Phenylketonuria (PKU) is a rare newborn genetic disease that impacts between 1 in 10,000 to 1 in 20,000 people, depending on the individuals' genetic ancestry. PKU causes an amino acid—called phenylalanine (Phe)—to build ...
Nov 2, 2023
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Medicine is at a turning point, on the cusp of major change as disruptive technologies such as gene, RNA, and cell therapies enable scientists to approach diseases in new ways. The swiftness of this change is being driven ...
Jan 10, 2019
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Babies born with a faulty maternal copy of the UBE3A gene will develop Angelman syndrome, a severe neurodevelopmental disorder with no cure and limited treatments. Now, for the first time, scientists at the UNC School of ...
Oct 21, 2020
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Researchers from Dana-Farber/Boston Children's Cancer and Blood Disorders Center have found that changes to a small stretch of DNA may circumvent the genetic defect behind sickle cell disease (SCD). The discovery, published ...
Sep 16, 2015
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Whether or not nerve cells are able to regrow after injury depends on their location in the body. Injured nerve cells in the peripheral nervous system, such as those in the arms and legs, can recover and regrow, at least ...
Feb 19, 2016
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Recent technological advances have revolutionised our ability to manipulate the genetic code, allowing us to specifically edit individual genes. Gene editing offers exciting potential for disease therapies but application ...
Dec 2, 2015
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