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          ﻿Title
                                                                              Disorder
                                                                              Modality
                                                                              PI/Lead Investigator
                                                                              InstitutionIND-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 CenterViral gene therapy for Menkes disease
                                                              Menkes Disease
                                                              Viral vector-based approaches (AAv9/ATP7A)
                                                              Stephen Gerard Kaler
                                                              The Research Institute at Nationside Childen's HospitalAdvancement 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 Current Research
  
    

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      Last reviewed on September 21, 2023
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