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A sickle cell first: Base editing, a new form of gene therapy, leaves patient feeling 'more than fine'
Though he doesn't remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got ...
Beam Therapeutics’ experimental gene-editing therapy for sickle cell disease now has its first cut of patient data showing signs of treating the rare blood disorder in a way that could differentiate ...
You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...
A schematic showing the cytosine base editing intermediate and the various outcomes that can occur when the UNG protein is active. You may have seen it in the news recently: a baby in Pennsylvania ...
Both CBEs and ABEs have been extensively engineered to improve their efficiency, specificity, and targeting range, enabling a wide variety of base pair conversions across the genome. Base editing can ...
While researchers have long appreciated the enormous potential of genome editing to treat disease, applications of these technologies in humans have historically been limited by safety concerns. Even ...
Researchers at the Broad Institute of MIT and Harvard have developed a gene-editing treatment for prion disease that extends lifespan by about 50 percent in a mouse model of the fatal ...
Development of bespoke therapies has been a driving goal in medical research. This year, the announcement of the first patient treated with personalized CRISPR therapy, Baby KJ, highlights this ...
Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Dr. David Liu, pictured with former lab members Holly Rees ...
Though he doesn’t remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got ...
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