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

Qure is planning soon to start enrolling patients in a second dose group as part of a clinical trial testing AMT-162 for SOD1 ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

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