A multi-institutional group of researchers led by Children's Hospital of Philadelphia (CHOP) has linked a strong cancer driver gene to changes in proteins that regulate alternative splicing. The researchers created new computational ...
Feb 17, 2020
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UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six ...
Jan 28, 2020
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276
Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers at Technical University of ...
Jan 28, 2020
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240
A single injection of a new gene therapy protected mice from the effects of sarin and other deadly chemical warfare agents for months, new work that could not only help defend soldiers and civilians in war zones, but also ...
Jan 23, 2020
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419
Researchers have found that melanoma cells fight anti-cancer drugs by changing their internal skeleton (cytoskeleton) - opening up a new therapeutic route for combatting skin and other cancers that develop resistance to treatment.
Jan 13, 2020
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Scientists at Scripps Research in Jupiter have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing.
Dec 24, 2019
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225
Anyone who climbs the 285 steps to the viewing platform of Berlin's Siegessäule, or Victory Column, will probably have quite a few sore muscles the next day. Out-of-the-ordinary activities such as climbing lots of steps ...
Dec 20, 2019
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55
Mount Sinai researchers have found that different mutations in a single gene can have myriad effects on a person's health, suggesting that gene therapies may need to do more than just replenish the missing or dysfunctional ...
Dec 18, 2019
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Scientists have developed a new gene-therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes.
Dec 17, 2019
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577
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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