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

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

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

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 ...

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

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

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

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

New gene therapy modalities, such as CRISPR guide RNA (single guide ribonucleic acid [sgRNA]) and messenger RNA (mRNA), ...

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