Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...
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The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...
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One shot to lower cholesterol for life? Scientists unlock a groundbreaking gene therapyResearchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...
Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...
The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
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