Current URGenT Projects

This is a preview of the Data Table. Only the first three rows are displayed.
Title Disorder Modality PI/Lead Investigator Institution
IND-enabling studies for Aspartylglucosaminuria (AGU) to support the initiation of an AAV9/AGA gene transfer clinical trial Aspartylglucosaminuria Viral vector-based approaches (AAV9/AGA) Steven J. Gray University of Texas Southwestern Medical Center
Viral gene therapy for Menkes disease Menkes Disease Viral vector-based approaches (AAv9/ATP7A) Stephen Gerard Kaler The Research Institute at Nationside Childen's Hospital
Advancement of Prion Protein-Lowering Divalent siRNA Therapy for Prion Disease Prion Disease Oligonucleotide-based approaches (siRNA) Eric Vallabh Minikel Broad Institute, Inc.
Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS Amyotrophic Lateral Sclerosis (ALS) Antisense Oligonucleotides (ASOs) Neil Alan Shneider Columbia University Health Sciences