A team of researchers from Intellia Therapeutics, Inc. and Regeneron Pharmaceuticals has conducted the first clinical trial involving in vivo CRISPR human gene editing. In their paper published in The New England Journal ...
The new drug sotorasib reduces tumor size and shows promise in improving survival among patients with lung tumors caused by a specific DNA mutation, according to results of a global phase 2 clinical trial. The drug is designed ...
Jun 4, 2021
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UNSW medical researchers have found a way to starve pancreatic cancer cells and 'disable' the cells that block treatment from working effectively. Their findings in mice and human lab models—which have been 10 years in ...
May 13, 2021
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An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National ...
May 12, 2021
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An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves ...
May 11, 2021
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A new gene therapy for one of the most common forms of congenital blindness was safe and improved patients' vision, according to initial data from a clinical trial led by researchers at the Scheie Eye Institute in the Perelman ...
Apr 16, 2021
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A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published today in eLife.
Apr 13, 2021
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For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, ...
Apr 9, 2021
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Results of a world-first Canadian pilot study on patients treated with gene therapy for Fabry disease show that the treatment is working and safe.
Feb 25, 2021
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Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons ...
Feb 19, 2021
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