Last Updated: 09/26/2026
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Found 7 publications
Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice.
Journal: The Journal of neuroscience : the official journal of the Society for Neuroscience
Published: October 03, 2022
Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH.
Journal: Molecular therapy. Methods & clinical development
Published: February 04, 2020
Adeno-Associated Virus Gene Therapy for Liver Disease.
Journal: Human gene therapy
Published: November 30, 2016
Safety Evaluation of CNS Administered Biologics-Study Design, Data Interpretation, and Translation to the Clinic.
Journal: Toxicological sciences : an official journal of the Society of Toxicology
Published: June 30, 2016
Workshop Proceedings: Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases-Are We There Yet?
Journal: International journal of toxicology
Published: June 09, 2016
Proof of concept for a banding scheme to support risk assessments related to multi-product biologics manufacturing.
Journal: Regulatory toxicology and pharmacology : RTP
Published: June 26, 2015
Showing 1-7 of 7
Last Updated: 09/26/2026