This page lists project-relevant scientific publications authored by the PaVe-GT project team.
2023
Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH PaVe-GT Experience
Richa M. Lomash, Oleg Shchelochkov, Randy J. Chandler, Charles P. Venditti, Anne Pariser, and Elizabeth Ottinger
2020
The Platform Vector Gene Therapies (PaVe-GT) Project: Increasing the efficiency of adeno-associated virus (AAV) gene therapy clinical trial startup
Philip J. Brooks, Elizabeth A. Ottinger, Deanna Portero, Richa Madan Lomash, Asaf Alimardanov, Pramod Terse, Xin Xu, Randy J. Chandler, Janelle Geist Hauserman, Eric Esposito, Carsten G. Bönnemann, Charles P. Venditti, Christopher P. Austin, Anne…
2019
Gene Therapy for Methylmalonic Acidemia: Past, Present and Future
Randy J. Chandler , Charles P Venditti
2016
Gene Therapy: The View from NCATS
Philip J. Brooks, N. Nora Yang, Christopher P. Austin
2010
Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia
Randy J. Chandler, Suma Chandrasekaran, Nuria Carrillo-Carrasco, Julien S. Senac, Sean E. Hofherr, Michael A. Barry, and Charles P. Venditti
Congenital Muscular Dystrophies: Toward Molecular Therapeutic Interventions
James Collins, Carsten G. Bönnemann
