Orphan Therapeutics Accelerator (OTXL), a patient-centered non-profit biotech focused on bringing promising clinical-stage ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...

Using X-ray diffraction data, James Watson (1928-2025) made the seminal discovery of the double-helical structure of DNA ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

Italian non-profit rare and complex genetic diseases specialist Fondazione Telethon (FT) and Orphan Therapeutics Accelerator ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

In a new study, scientists at the Max Planck Institute for Evolutionary Anthropology in Leipzig analyzed the impact of more ...

To our immune system, a potentially lifesaving gene therapy can look a lot like a dangerous infection. That's because most ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

Researchers at the University of Geneva, together with colleagues in Switzerland, France, the United States and Israel, ...