Sentences with phrase «new type of gene therapy»
Not exact matches
A one - time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published in the New England Journal of Medicine.
https://www.sciencedaily.com/
The new method may make some types of gene therapy easier and could be a boon for researchers hoping to control gene activity in animals, scientists say.
The new method may make some types of gene therapy easier...
April 23, 2018 Ultragenyx Announces Filing and FDA Clearance of an Investigational New Drug Application for DTX401, a Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia
«The ability to direct a gene to a specific cell type and prevent expression in other cell types is a powerful new tool that allows us to bypass one of the most troubling safety concerns facing gene therapy,» said Michael Parmacek, MD, assistant professor of medicine at the University of Chicago and director of the study.
«This new work is exciting, because it shows that we can directly target genes within a specific cell type, which is important for targeted therapies,» says Hedrick, a Professor in the Division of Inflammation Biology.