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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20
In a landmark study that could lead to new therapies for sickle cell anaemia and other blood disorders, UNSW Sydney-led researchers have used CRISPR gene editing to introduce beneficial natural mutations into blood cells ...
Apr 2, 2018
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Scientists at Cold Spring Harbor Laboratory (CSHL) have discovered a way to rein in an overactive protein that drives some aggressive leukemias. The renegade molecule, MEF2C, belongs to a class of proteins that is notoriously ...
Mar 8, 2018
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For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that made this possible.
Feb 13, 2018
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78
ALS and frontotemporal dementia (FTD) are two neurodegenerative diseases with a toxic relationship, according to a new USC Stem Cell study published in Nature Medicine.
Feb 5, 2018
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127
After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping ...
Dec 28, 2017
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426
After years of rigorous research, a team of scientists has identified the genetic engine that drives a rare form of liver cancer. The findings offer prime targets for drugs to treat the usually lethal disease, fibrolamellar ...
Nov 21, 2017
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Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to try to cure a disease.
Nov 15, 2017
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1117
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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The public should be consulted before gene editing is used to treat human embryos, according to a survey of scientists published in the American Heart Association's journal Circulation: Cardiovascular Genetics.
Oct 3, 2017
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