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

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

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

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 ...

McDonough. More: Anne Frank exhibition on display at Raritan Valley Community College in Branchburg The cell and gene therapy ...

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

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

TCTMD ® is produced by the Cardiovascular Research Foundation ® (CRF). CRF ® is committed to igniting the next wave of innovation in research and education that will help doctors save and improve the ...

The five-year trial saw patients who received a single injection of Lumevoq sustaining clinical recovery in a rare condition ...

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