gene therapy
Fetal Gene Therapy Prevents Gaucher Disease
Using gene therapy on the brains of fetal mice and non-human primates, researchers have managed to prevent a fatal neurodegenerative condition known as Gaucher disease.
Gene Therapy Reduces Heart Failure In Mice
By inhibiting the production of ANGPTL2, researchers were able to reduce the progression of heart failure in mouse models.
Treating Huntington’s With Gene Knockout Might Be Safe For Adults
Although deleting the huntingtin gene is lethal for young mice, it appears to safely treat the disease in adult mice.
Delivering mRNA To Treat Neurological Diseases
By protecting the mRNA in a nanopolymer, scientists have opened up the possibility of using mRNA for gene therapy.
Astellas & Harvard Take On Retinitis Pigmentosa
Astellas will fund a study of genetic retinal degenerative disease run by Dr. Constance Cepko from Harvard University.
