First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial

20250 citationsJournal Articlegold Open Access

Authors

Benjamin Greenberg

Berge A. Minassian · The University of Texas Southwestern Medical Center

Souad Messahel · Poseida Therapeutics (United States)

Veronica Bordes Edgar · The University of Texas Southwestern Medical Center

Andrea Lowden · The University of Texas Southwestern Medical Center

Hamza Dahshi · Case Western Reserve University

Emily R. Nettesheim · The University of Texas Southwestern Medical Center

Abstract

This study was funded by The Batten's Hope Foundation, Mila's Miracle Foundation, Children's Health Dallas and Philanthropic Gifts to UT Southwestern. In addition, Emily R. Nettesheim received funding from NIH training grant 5T32GM131945-03 and Hamza Dahshi was supported in part by NIH award T32 GM152319.

Topics & Keywords

Virus-based gene therapy research Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus

Publication Details

Published in: EBioMedicine

Volume 123, pp. 106044-106044

DOI: 10.1016/j.ebiom.2025.106044

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First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial

Authors

Abstract

Topics & Keywords

Publication Details