E. Shyam P. Reddy: First CRISPR gene repair in the human body shows promising results
E. Shyam P. Reddy, Professor and Director of the Cancer Biology Program, Dept OB/GYN at Morehouse School of Medicine, shared a post on LinkedIn:
“For the first time, researchers have used the CRISPR gene editor to repair a flawed gene in the human body. In a press release, the biotechnology company Beam Therapeutics announced it had given nine patients with a lung and liver disease called alpha-1 antitrypsin (AAT) deficiency an intravenous infusion of lipid nanoparticles.
These particles contained instructions that let the body’s liver cells make a version of CRISPR designed to correct a single mutated base in a gene that produces the AAT protein.
In people with AAT deficiency, the uncorrected gene causes the body to produce a flawed, toxic version of the AAT protein that harms the lungs’ ability to deal with inflammation and builds up in the liver.
After receiving the experimental infusion, the patients’ blood levels of the corrected protein rose while levels of the flawed protein dropped, in some cases enough that liver and lung damage should be halted, The New York Times reports.
So-called base editing has previously been used in the liver for a heart disease, but to disable a gene and not repair it, in a trial that ran into safety problems. Beam now plans to test higher doses of its treatment.”
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