Search results

7 items matching your search terms.
Filter the results.
Item type













New items since



Sort by relevance · date (newest first) · alphabetically
Charlotte Charity Inspires New Law Designed to Spur Treatments for Rare Diseases and Economic Growth for North Carolina
Law Will Establish an Advisory Council on Rare Diseases at the University of North Carolina at Chapel Hill
Located in News / 2015 / August
New gene therapy proves promising as hemophilia treatment
Researchers package specialized blood platelets with genes that express clotting factor, leading to fewer bleeding events.
Located in News / 2013 / December
Preliminary Data from Groundbreaking Genome Editing Clinical Trial Encouraging
As part of the phase 1 clinical trial for patients with Hunter syndrome, Joseph Muenzer, MD, PhD, performed the UNC Clinical Translational and Research Center's first-ever in vivo genome editing therapy, achieving a milestone in his own career.
Located in News / 2018 / September
Zeng wins Outstanding Individual Performance Award
Allergen Biologics created this award due to its first-rate relationship with Xinghua Zeng, the lab manager for the UNC Vector Core, which provides AAV products to researchers around the world.
Located in News / 2019 / February
Newly designed viral vectors could lead to improved gene therapies
UNC and University of Florida researchers created viruses to deliver gene therapies while evading pre-existing immune system responses. Aravind Asokan, PhD, led the research team at UNC.
Located in News / 2017 / June
Promising results with new gene therapy approach for treating inherited neurodegenerative diseases
Approach developed in part by UNC’s Dr. Steven Gray restored enzyme function and extended survival time in treated mice.
Located in News / 2016 / July
Children's Research Institute Seminar Series: "Cross-blood-brain-barrier Gene Delivery for Treating Neuropathic Lysosomal Storage Diseases"
The Children’s Research Institute Seminar Series presents its September speaker, Haiyan Fu, PhD, for her talk, "Cross-blood-brain-barrier Gene Delivery for Treating Neuropathic Lysosomal Storage Diseases."
Located in Vital Signs / 2018 / Sept. 6