Latest Human Gene Therapy Stories
Gene therapy strategies to prevent and treat inherited diseases of the retina that can cause blindness have progressed rapidly.
A recent article in the New England Journal of Medicine showed that Factor IX gene therapy in patients with hemophilia B was able to convert severe hemophilia to moderate or even mild disease.
The introduction of a new, fully characterized viral vector for use as reference material to help standardize gene therapy protocols in research applications and human clinical trials is described in an article in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com).
The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit.
- The horn of a unicorn considered as a medical or pharmacological ingredient.
- A winged horse with a single horn on its head; a winged unicorn.