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Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...

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 ...

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, ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

The Metachromatic Leukodystrophy Initiative (MLDi) critically responds to the findings of Zhang et al. on lentivirus-modified ...

Older women could be vulnerable to harmful inflammation from new gene therapies to treat incurable eye diseases, new research has found.