A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led by researchers at The Ohio State University ...
Jul 12, 2021
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Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Genethon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of ...
Jun 28, 2021
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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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